Menu UF Health Home Menu
 

Recent Publications

2016 Center Research

  • Ash J. D. (2016) The Potential Use of PGC-1α and PGC-1β to Protect the Retina by Stimulating Mitochondrial Repair. Retinal Degenerative Diseases. C. Bowes Rickman, M. M. LaVail, R. E. Anderson et al., Springer International Publishing. 854: 403-409.
  • Ahmed C. M., Biswal M. R., Li H., Han P., Ildefonso C. J., Lewin A. S. (2016) “Repurposing an orally available drug for the treatment of geographic atrophy.” Mol Vis 22: 294-310.
  • Beal C., Giordano B. (2016) “Clinical evaluation of red eyes in pediatric patients.” J Pediatr Health Care 30: 506-514.
  • Berkowitz B. A., Lewin A. S., Biswal M. R., Bredell B. X., Davis C., Roberts R. (2016) “MRI of retinal free radical production with laminar resolution in vivo.” Invest Ophthalmol Vis Sci 57: 577-585.
  • Bolch S. N., Dugger D. R., Chong T., McDowell J. H., Smith W. C. (2016) “A splice variant of Bardet-Biedl Syndrome 5 (BBS5) protein that is selectively expressed in retina.” PLoS ONE 11: e0148773.
  • Boyd R. F., Boye S. L., Conlon T. J., Erger K. E., Sledge D. G., Langohr I. M., Hauswirth W. W., Komaromy A. M., Boye S. E., Petersen-Jones S. M., Bartoe J. T. (2016) “Reduced retinal transduction and enhanced transgene-directed immunogenicity with intravitreal delivery of rAAV following posterior vitrectomy in dogs.” Gene Ther 23: 548-556.
  • Boyd R. F., Sledge D. G., Boye S. L., Boye S. E., Hauswirth W. W., Komaromy A. M., Petersen-Jones S. M., Bartoe J. T. (2016) “Photoreceptor-targeted gene delivery using intravitreally administered AAV vectors in dogs.” Gene Ther 23: 223-230.
  • Boye S. E., Alexander J. J., Witherspoon C. D., Boye S. L., Peterson J. J., Clark M. E., Sandefer K. J., Girkin C. A., Hauswirth W. W., Gamlin P. D. (2016) “Highly efficient delivery of adeno-associated viral vectors to the primate retina.” Human Gene Ther 27: 580-597.
  • Boye S. L., Bennett A., Scalabrino M. L., McCullough K. T., Van Vliet K., Choudhury S., Ruan Q., Peterson J., Agbandje-McKenna M., Boye S. E. (2016) “Impact of heparan sulfate binding on transduction of retina by recombinant adeno-associated virus vectors.” J Virol 90: 4215-4231.
  • Boye S. L., Olshevskaya E. V., Peshenko I. V., McCullough K. T., Boye S. E., Dizhoor A. M. (2016) “Functional study of two biochemically unusual mutations in GUCY2D Leber congenital amaurosis expressed via adenoassociated virus vector in mouse retinas.” Mol Vis 22: 1342-1351.
  • Choudhury S., Strang C., Alexander J., Scalabrino M., Lynch Hill J., Kasuga D., Witherspoon C., Boye S., Gamlin P., Boye S. (2016) “Novel Methodology for Creating Macaque Retinas with Sortable Photoreceptors and Ganglion Cells.” Frontiers in Neuroscience 10.
  • Dinculescu A., Stupay R. M., Deng W.-T., Dyka F. M., Min S.-H., Boye S. L., Chiodo V. A., Abrahan C. E., Zhu P., Li Q., Strettoi E., Novelli E., Nagel-Wolfrum K., Wolfrum U., Smith W. C., Hauswirth W. W. (2016) “AAV-mediated Clarin-1 expression in the mouse retina: implications for USH3A gene therapy.” PLoS ONE 11: e0148874.
  • Dominguez Ii J. M., Hu P., Caballero S., Moldovan L., Verma A., Oudit G. Y., Li Q., Grant M. B. (2016) “Adeno-associated virus overexpression of angiotensin-converting enzyme-2 reverses diabetic retinopathy in Type 1 diabetes in mice.” Am J Pathol 186: 1688-1700.
  • Ghazi N. G., Abboud E. B., Nowilaty S. R., Alkuraya H., Alhommadi A., Cai H., Hou R., Deng W.-T., Boye S. L., Almaghamsi A., Al Saikhan F., Al-Dhibi H., Birch D., Chung C., Colak D., LaVail M. M., Vollrath D., Erger K., Wang W., Conlon T., Zhang K., Hauswirth W., Alkuraya F. S. (2016) “Treatment of retinitis pigmentosa due to MERTK mutations by ocular subretinal injection of adeno-associated virus gene vector: results of a phase I trial.” Human Genetics 135: 327-343.
  • Giordano B. P., Tuli S. S., Ryan S. F., Stern M., Tuli S. Y. (2016) “Crouzon Syndrome: Visual diagnosis.” J Pediatr Health Care 30: 270-273.
  • Hickmott J. W., Chen C.-y., Arenillas D. J., Korecki A. J., Lam S. L., Molday L. L., Bonaguro R. J., Zhou M., Chou A. Y., Mathelier A., Boye S. L., Hauswirth W. W., Molday R. S., Wasserman W. W., Simpson E. M. (2016) “PAX6 minipromoters drive restricted expression from rAAV in the adult mouse retina.” Mol Ther Methods Clin Dev 3: 16051.
  • Ildefonso C. J., Jaime H., Brown E. E., Iwata R. L., Ahmed C. M., Massengill M. T., Biswal M. R., Boye S. E., Hauswirth W. W., Ash J. D., Li Q., Lewin A. S. (2016) “Targeting the Nrf2 signaling pathway in the retina with a gene-delivered secretable and cell-penetrating peptide.” Invest Ophthalmol Vis Sci 57: 372-386.
  • Jara J. H., Stanford M. J., Zhu Y., Tu M., Hauswirth W. W., Bohn M. C., DeVries S. H., Ozdinler P. H. (2016) “Healthy and diseased corticospinal motor neurons are selectively transduced upon direct AAV2-2 injection into the motor cortex.” Gene Ther 23: 272-282.
  • Langlo C. S., Patterson E. J., Higgins B. P., Summerfelt P., Razeen M. M., Erker L. R., Parker M., Collison F. T., Fishman G. A., Kay C. N., Zhang J., Weleber R. G., Yang P., Wilson D. J., Pennesi M. E., Lam B. L., Chiang J., Chulay J. D., Dubra A., Hauswirth W. W., Carroll J. (2016) “Residual foveal cone structure in CNGB3-associated achromatopsia.” Invest Ophthalmol Vis Sci 57: 3984-3995.
  • Langouet-Astrie C. J., Yang Z., Polisetti S. M., Welsbie D. S., Hauswirth W. W., Zack D. J., Merbs S. L., Enke R. A. (2016) “Characterization of intravitreally delivered capsid mutant AAV2-Cre vector to induce tissue-specific mutations in murine retinal ganglion cells.” Exp Eye Res 151: 61-67.
  • Liu X., Dreffs A., Díaz-Coránguez M., Runkle E. A., Gardner T. W., Chiodo V. A., Hauswirth W. W., Antonetti D. A. (2016) “Occludin S490 phosphorylation regulates vascular endothelial growth factor–induced retinal neovascularization.” Am J Pathol 186: 2486-2499.
  • Liu X Z. Y., Du W, Shi W, Tao Y, Deng W, Li J, Zhao C, Pang J (2016) “Evaluation of AAV-mediated gene therapy with reduced vector volume in Cngb3 knockout mice, a model of achromatopsia.” Hereditary Genetics 5.
  • Qiu Y., Tao L., Zheng S., Lin R., Fu X., Chen Z., Lei C., Wang J., Li H., Li Q., Lei B. (2016) “AAV8-mediated angiotensin-converting enzyme 2 gene delivery prevents experimental autoimmune uveitis by regulating MAPK, NF-κB and STAT3 pathways.” Scientific Reports 6: 31912.
  • Rex T. S., Kasmala L., Bond W. S., de Lucas Cerrillo A. M., Wynn K., Lewin A. S. (2016) “Erythropoietin slows photoreceptor cell death in a mouse model of autosomal dominant retinitis pigmentosa.” PLoS ONE 11: e0157411.
  • Schaefer J. L., Lukowski Z. L., Meyer A. M., Leoncavallo A. J., Greer A., Martorana G. M., Zou B., Shuster J. J., Sherwood M. B. (2016) “Comparing glaucomatous disc change using stereo disc viewing and the MatchedFlicker software program in Ophthalmologists-in-Training.” Am J Ophthalmol 167: 88-95.
  • Tezel G., Hernandez M., Wax M. B. (2000) “IMmunostaining of heat shock proteins in the retina and optic nerve head of normal and glaucomatous eyes.” Archives of Ophthalmology 118: 511-518.
  • Tuli S., Gray M. (2016) “Surgical management of corneal infections.” Curr Opin Ophthalmol 27: 340–347.
  • Watson Z. L., Ertel M. K., Lewin A. S., Tuli S. S., Schultz G. S., Neumann D. M., Bloom D. C. (2016) “Adeno-associated virus vectors efficiently transduce mouse and rabbit sensory neurons coinfected with Herpes Simplex Virus 1 following peripheral inoculation.” J Virol 90: 7894-7901.
  • Weleber R. G., Pennesi M. E., Wilson D. J., Kaushal S., Erker L. R., Jensen L., McBride M. T., Flotte T. R., Humphries M., Calcedo R., Hauswirth W. W., Chulay J. D., Stout J. T. (2016) “Results at 2 years after gene therapy for RPE65-deficient Leber congenital amaurosis and severe early-childhood–onset retinal dystrophy.” Ophthalmol 123: 1606-1620.
  • Xie K., Colgan Lesley A., Dao Maria T., Muntean Brian S., Sutton Laurie P., Orlandi C., Boye Sanford L., Boye Shannon E., Shih C.-C., Li Y., Xu B., Smith Roy G., Yasuda R., Martemyanov Kirill A. “NF1 Is a direct g protein effector essential for opioid signaling to Ras in the striatum.” Curr Biol.
  • Yan B., Vakulenko M., Min S.-H., Hauswirth W. W., Nirenberg S. (2016) “Maintaining ocular safety with light exposure, focusing on devices for optogenetic stimulation.” Vis Res 121: 57-71.
  • Yang F., Ma H., Belcher J., Butler M. R., Redmond T. M., Boye S. L., Hauswirth W. W., Ding X.-Q. (2016) “Targeting iodothyronine deiodinases locally in the retina is a therapeutic strategy for retinal degeneration.” FASEB J.
  • Yang H., Zheng S., Mao Y., Chen Z., Zheng C., Li H., Sumners C., Li Q., Yang P., Lei B. (2016) “Modulating of ocular inflammation with macrophage migration inhibitory factor is associated with notch signalling in experimental autoimmune uveitis.” Clin Exp Immunol 183: 280-293.
  • Ye G.-j., Budzynski E., Sonnentag P., Nork T. M., Miller P. E., McPherson L., Ver Hoeve J. N., Smith L. M., Arndt T., Mandapati S., Robinson P. M., Calcedo R., Knop D. R., Hauswirth W. W., Chulay J. D. (2016) “Safety and biodistribution evaluation in CNGB3-Deficient Mice of rAAV2tYF-PR1.7-hCNGB3, a recombinant aav vector for treatment of achromatopsia.” Hum Gene Ther Clin Dev 27: 27-36.
  • Ye G.-j., Budzynski E., Sonnentag P., Nork T. M., Miller P. E., Sharma A. K., Ver Hoeve J. N., Smith L. M., Arndt T., Calcedo R., Gaskin C., Robinson P. M., Knop D. R., Hauswirth W. W., Chulay J. D. (2016) “Safety and biodistribution evaluation in cynomolgus macaques of rAAV2tYF-PR1.7-hCNGB3, a recombinant aav vector for treatment of achromatopsia.” Hum Gene Ther Clin Dev 27: 37-48.
  • Ye G.-J., Budzynski E., Sonnentag P., Nork T. M., Sheibani N., Gurel Z., Boye S. L., Peterson J. J., Boye S. E., Hauswirth W. W., Chulay J. D. (2015) “Cone-specific promoters for gene therapy of achromatopsia and other retinal diseases.” Hum Gene Ther 27: 72-82.
  • Ying G., Gerstner C. D., Frederick J. M., Boye S. L., Hauswirth W. W., Baehr W. (2016) “Small GTPases Rab8a and Rab11a are dispensable for rhodopsin transport in mouse photoreceptors.” PLoS ONE 11: e0161236.

2015 Center Research

  • Ash J. D. (2016) The Potential Use of PGC-1α and PGC-1β to Protect the Retina by Stimulating Mitochondrial Repair. Retinal Degenerative Diseases. C. Bowes Rickman, M. M. LaVail, R. E. Anderson et al., Springer International Publishing. 854: 403-409.
  • Banin E., Gootwine E., Obolensky A., Ezra-Elia R., Ejzenberg A., Zelinger L., Honig H., Rosov A., Yamin E., Sharon D., Averbukh E., Hauswirth W. W., Ofri R. (2015) “Gene augmentation therapy restores retinal function and visual behavior in a sheep model of CNGA3 achromatopsia.” Mol Ther 23: 1423-1433.
  • Battelle B.-A., Kempler K. E., Saraf S. R., Marten C. E., Dugger D. R., Speiser D. I., Oakley T. H. (2015) “Opsins in Limulus eyes: characterization of three visible light-sensitive opsins unique to and co-expressed in median eye photoreceptors and a peropsin/RGR that is expressed in all eyes.” J Exp Biol 218: 466-479.
  • Beltran W. A., Cideciyan A. V., Iwabe S., Swider M., Kosyk M. S., McDaid K., Martynyuk I., Ying G.-S., Shaffer J., Deng W.-T., Boye S. L., Lewin A. S., Hauswirth W. W., Jacobson S. G., Aguirre G. D. (2015) “Successful arrest of photoreceptor and vision loss expands the therapeutic window of retinal gene therapy to later stages of disease.” Proceedings of the National Academy of Sciences 112: E5844-E5853.
  • Benskey M. J., Kuhn N. C., Galligan J. J., Garcia J., Boye S. E., Hauswirth W. W., Mueller C., Boye S. L., Manfredsson F. P. (2015) “Targeted gene delivery to the enteric nervous system using AAV: A comparison across serotypes and capsid mutants.” Mol Ther 23: 488-500.
  • Bogner B., Boye S. L., Min S. H., Peterson J. J., Ruan Q., Zhang Z., Reitsamer H. A., Hauswirth W. W., Boye S. E. (2015) “Capsid mutated adeno-associated virus delivered to the anterior chamber results in efficient transduction of trabecular meshwork in mouse and rat.” PLoS ONE 10: e0128759.
  • Boyd R. F., Sledge D. G., Boye S. L., Boye S. E., Hauswirth W. W., Komaromy A. M., Petersen-Jones S. M., Bartoe J. T. (2015) “Photoreceptor-targeted gene delivery using intravitreally administered AAV vectors in dogs.” Gene Ther.
  • Boye S. E. (2016) A Mini-review: Animal Models of GUCY2D Leber Congenital Amaurosis (LCA1). Retinal Degenerative Diseases. C. Bowes Rickman, M. M. LaVail, R. E. Anderson et al., Springer International Publishing. 854: 253-258.
  • Boye S. L., Peterson J. J., Choudhury S., Min S. H., Ruan Q., McCullough K. T., Zhang Z., Olshevskaya E. V., Peshenko I. V., Hauswirth W. W., Ding X.-Q., Dizhoor A. M., Boye S. E. (2015) “Gene therapy fully restores vision to the all-cone Nrl−/−Gucy2e−/− mouse model of leber congenital amaurosis-1.” Hum Gene Ther 26: 575-592.
  • Chen H., Zheng D., Ambadapadi S., Davids J., Ryden S., Samy H., Bartee M., Sobel E., Dai E., Liu L., Macaulay C., Yachnis A., Weyand C., Thoburn R., Lucas A. (2015) “Serpin Treatment Suppresses Inflammatory Vascular Lesions in Temporal Artery Implants (TAI) from Patients with Giant Cell Arteritis.” PLoS ONE 10: e0115482.
  • Dai X., Zhang H., He Y., Qi Y., Chang B., Pang J.-j. (2015) “The frequency-response electroretinogram distinguishes cone and abnormal rod function in rd12 mice.” PLoS ONE 10: e0117570.
  • Deng W.-T., Dyka F. M., Dinculescu A., Li J., Zhu P., Chiodo V. A., Boye S. L., deng T. J., Erger K., Cossette T., Hauswirth W. W. (2015) “Stability and safety of an AAV vector for treating RPGR-ORF15 X-linked retinitis pigmentosa.” Hum Gene Ther 26: 593-602.
  • Dinculescu A., Min S.-H., Dyka F. M., Deng W.-T., Stupay R. M., Chiodo V., Smith W. C., Hauswirth W. W. (2015) “Pathological effects of mutant C1QTNF5 (S163R) expression in murine retinal pigment epitheliumpathological effects of mutant C1QTNF5 (S163R).” Invest Ophthalmol Vis Sci 56: 6971-6980.
  • Du W., Tao Y., Deng W.-T., Zhu P., Li J., Dai X., Zhang Y., Shi W., Liu X., Chiodo V. A., Ding X.-Q., Zhao C., Michalakis S., Biel M., Zhang Z., Qu J., Hauswirth W. W., Pang J.-j. (2015) “Vitreal delivery of AAV vectored Cnga3 restores cone function in CNGA3−/−/Nrl−/− mice, an all-cone model of CNGA3 achromatopsia.” Hum Mol Genet 24: 3699-3707.
  • Giordano B. P., Tuli S. S., Ryan S. F., Stern M., Tuli S. Y. “Crouzon syndrome: visual diagnosis.” J Ped Health Care.
  • Ildefonso C. J., Jaime H., Biswal M. R., Boye S. E., Li Q., Hauswirth W. W., Lewin A. S. (2015) “Gene therapy with the caspase activation and recruitment domain reduces the ocular inflammatory response.” Mol Ther 23: 875-884.
  • Ildefonso C. J., Jaime H., Rahman M. M., Li Q., Boye S. E., Hauswirth W. W., Lucas A. R., McFadden G., Lewin A. S. (2015) “Gene delivery of a viral anti-inflammatory protein to combat ocular inflammation.” Hum Gene Ther 26: 59-68.
  • Jacobson S. G., Cideciyan A. V., Roman A. J., Sumaroka A., Schwartz S. B., Heon E., Hauswirth W. W. (2015) “Improvement and decline in vision with gene therapy in childhood blindness.” New Engl J Med 372: 1920-1926.
  • Jiang L., Tam B. M., Ying G., Wu S., Hauswirth W. W., Frederick J. M., Moritz O. L., Baehr W. (2015) “Kinesin family 17 (osmotic avoidance abnormal-3) is dispensable for photoreceptor morphology and function.” FASEB J.
  • Kelly M. N., Tuli S. Y., Tuli S. S., Stern M. A., Giordano B. P. (2015) “Brothers with Smith-Lemli-Opitz syndrome.” J Ped Health Care 29: 97-103.
  • Ku C. A., Chiodo V. A., Boye S. L., Hayes A., Goldberg A. F. X., Hauswirth W. W., Ramamurthy V. (2015) “Viral-mediated vision rescue of a novel AIPL1 cone-rod dystrophy model.” Hum Molec Genet 24: 670-684.
  • Kwong J. M. K., Gu L., Nassiri N., Bekerman V., Kumar-Singh R., Rhee K. D., Yang X. J., Hauswirth W. W., Caprioli J., Piri N. (2015) “AAV-mediated and pharmacological induction of Hsp70 expression stimulates survival of retinal ganglion cells following axonal injury.” Gene Ther 22: 138-145.
  • LaVail M. M., Yasumura D., Matthes M. T., Yang H., Hauswirth W. W., Deng W.-T., Vollrath D. (2016) Gene Therapy for MERTK-Associated Retinal Degenerations. Retinal Degenerative Diseases. C. Bowes Rickman, M. M. LaVail, R. E. Anderson et al., Springer International Publishing. 854: 487-493.
  • Li J. B., Gerdes J. M., Haycraft C. J., Fan Y., Teslovich T. M., May-Simera H., Li H., Blacque O. E., Li L., Leitch C. C., Lewis R. A., Green J. S., Parfrey P. S., Leroux M. R., Davidson W. S., Beales P. L., Guay-Woodford L. M., Yoder B. K., Stormo G. D., Katsanis N., Dutcher S. K. (2004) “Comparative Genomics Identifies a Flagellar and Basal Body Proteome that Includes the BBS5 Human Disease Gene.” Cell 117: 541-552.
  • Lipinski D. M., Reid C. A., Boye S. L., Peterson J. J., Qi X., Boye S. E., Boulton M. E., Hauswirth W. W. (2015) “Systemic vascular transduction by capsid mutant adeno-associated virus after intravenous injection.” Hum Gene Ther 26: 767-776.
  • Martorana G. M., Schaefer J. L., Levine M. A., Lukowski Z. L., Min J., Meyers C. A., Schultz G. S., Sherwood M. B. (2015) “Sequential therapy with saratin, bevacizumab and ilomastat to prolong bleb function following glaucoma filtration surgery in a rabbit model.” PLoS ONE 10: e0138054.
  • Patel S., Fargen K. M., Peters K., Krall P., Samy H., Hoh B. L. (2015) “Return of visual function after bilateral visual loss following flow diversion embolization of a giant ophthalmic aneurysm due to both reduction in mass effect and reduction in aneurysm pulsation.” Journal of NeuroInterventional Surgery 7: e1.
  • Qi Y., Dai X., Zhang H., He Y., Zhang Y., Han J., Zhu P., Zhang Y., Zheng Q., Li X., Zhao C., Pang J. (2015) “Trans-corneal subretinal injection in mice and its effect on the function and morphology of the retina.” PLoS ONE 10: e0136523.
  • Qiu Y., Tao L., Lei C., Wang J., Yang P., Li Q., Lei B. (2015) “Downregulating p22phox ameliorates inflammatory response in Angiotensin II-induced oxidative stress by regulating MAPK and NF-κB pathways in ARPE-19 cells.” Scientific Reports 5: 14362.
  • Reagan A., Gu X., Hauck S. M., Ash J. D., Cao G., Thompson T. C., Elliott M. H. (2016) Retinal Caveolin-1 Modulates Neuroprotective Signaling. Retinal Degenerative Diseases. C. Bowes Rickman, M. M. LaVail, R. E. Anderson et al., Springer International Publishing. 854: 411-418.
  • Scalabrino M. L., Boye S. L., Fransen K. M. H., Noel J. M., Dyka F. M., Min S. H., Ruan Q., De Leeuw C. N., Simpson E. M., Gregg R. G., McCall M. A., Peachey N. S., Boye S. E. (2015) “Intravitreal delivery of a novel AAV vector targets ON bipolar cells and restores visual function in a mouse model of complete congenital stationary night blindness.” Hum Molec Genet 24: 6229-6239.
  • Schaefer J. L., Levine M. A., Martorana G., Koenigsman H., Smith M. F., Sherwood M. B. (2015) “Failed glaucoma drainage implant: long-term outcomes of a second glaucoma drainage device versus cyclophotocoagulation.” Br J Ophthalmol.
  • Shu X., Pang J.-j., Zhang H., Mansfield D. (2015) “Retinitis pigmentosa: Disease mechanisms, diagnosis, and therapies.” J Ophthalmol 2015: 1.
  • Talla V., Koilkonda R., Porciatti V., Chiodo V., Boye S. L., Hauswirth W. W., Guy J. (2015) “Complex I subunit gene therapy with NDUFA6 ameliorates neurodegeneration in EAE Complex I subunit gene therapy in EAE.” Invest Ophthalmol Vis Sci 56: 1129-1140.
  • Thompson D. A., Ali R. R., Banin E., Branham K. E., Flannery J. G., Gamm D. M., Hauswirth W. W., Heckenlively J. R., Iannaccone A., Jayasundera K. T., Khan N. W., Molday R. S., Pennesi M. E., Reh T. A., Weleber R. G., Zacks D. N. (2015) “Advancing Therapeutic strategies for inherited retinal degeneration: Recommendations from the Monaciano Symposium Advancing Therapies for Retinal Dystrophies.” Invest Ophthalmol Vis Sci 56: 918-931.
  • Xu L., Ash J. D. (2016) The Role of AMPK Pathway in Neuroprotection. Retinal Degenerative Diseases. C. Bowes Rickman, M. M. LaVail, R. E. Anderson et al., Springer International Publishing. 854: 425-430.
  • Yang H., Zheng S., Mao Y., Chen Z., Zheng C., Li H., Sumners C., Li Q., Yang P., Lei B. (2015) “Modulating of ocular inflammation with macrophage migration inhibitory factor is associated with Notch signaling in experimental autoimmune uveitis.” Clin Exp Immunol: n/a-n/a.
  • Yu H., Koilkonda R. D., Chou T.-H., Porciatti V., Mehta A., Hentall I. D., Chiodo V. A., Boye S. L., Hauswirth W. W., Lewin A. S., Guy J. (2015) “Consequences of zygote injection and germline transfer of mutant human mitochondrial DNA in mice.” Proc Natl Acad Sci USA 112: E5689-E5698.
  • Zhang H., Dai X., Qi Y., He Y., Du W., Pang J.-j. (2015) “Histone deacetylases inhibitors in the treatment of retinal degenerative diseases: Overview and perspectives.” J Ophthalmol 2015: 9.
  • Zheng Q., Ren Y., Tzekov R., Hua S., Li M., Pang J., Qu J., Li W. (2015) “iTRAQ-based proteomic analysis of visual cycle-associated proteins in RPE of rd12 mice before and after RPE65 gene delivery.” J Ophthalmol 2015: 8.
  • Zhong H., Eblimit A., Moayedi Y., Boye S. L., Chiodo V. A., Chen Y., Li Y., Nichols R. M., Hauswirth W. W., Chen R., Mardon G. (2015) “AAV8(Y733F)-mediated gene therapy in a Spata7 knockout mouse model of Leber congenital amaurosis and retinitis pigmentosa.” Gene Ther 22: 619-627.

2014 Center Research

  • Batenburg W. W., Verma A., Wang Y., Zhu P., van den Heuvel M., van Veghel R., Danser A. H. J., Li Q. (2014) “Combined Renin Inhibition/(Pro)Renin Receptor Blockade in Diabetic Retinopathy- A Study in Transgenic (mREN2)27 Rats.” PLoS ONE 9: e100954.
  • Battelle B.-A., Kempler K. E., Harrison A., Dugger D. R., Payne R. (2014) “Opsin expression in Limulus eyes: a UV opsin is expressed in each eye type and co-expressed with a visible light-sensitive opsin in ventral larval eyes.” The Journal of Experimental Biology 217: 3133-3145.
  • Beltran W. A., Cideciyan A. V., Lewin A. S., Hauswirth W. W., Jacobson S. G., Aguirre G. D. (2014) “Gene Augmentation for X-Linked Retinitis Pigmentosa Caused by Mutations in RPGR.” Cold Spring Harbor Perspectives in Medicine.
  • Boye S. E. (2014) “Insights gained from gene therapy in animal models of retGC1 deficiency.” Frontiers in Molecular Neuroscience 7.
  • Boye S. E. (2014) “Leber Congenital Amaurosis Caused by Mutations in GUCY2D.” Cold Spring Harbor Perspectives in Medicine.
  • Boye S. E., Huang W.-C., Roman A. J., Sumaroka A., Boye S. L., Ryals R. C., Olivares M. B., Ruan Q., Tucker B. A., Stone E. M., Swaroop A., Cideciyan A. V., Hauswirth W. W., Jacobson S. G. (2014) “Natural History of Cone Disease in the Murine Model of Leber Congenital Amaurosis Due to <italic>CEP290</italic> Mutation: Determining the Timing and Expectation of Therapy.” PLoS ONE 9: e92928.
  • Dai X., Han J., Qi Y., Zhang H., Xiang L., Lv J., Li J., Deng W.-T., Chang B., Hauswirth W. W., Pang J.-j. (2014) “AAV-Mediated Lysophosphatidylcholine Acyltransferase 1 (Lpcat1) Gene Replacement Therapy Rescues Retinal Degeneration in rd11 Mice.” Investigative Ophthalmology & Visual Science 55: 1724-1734.
  • de Leeuw C. N., Dyka F. M., Boye S. L., Laprise S., Zhou M., Chou A. Y., Borretta L., McInerny S. C., Banks K. G., Portales-Casamar E., Swanson M. I., D’Souza C. A., Boye S. E., Jones S. J., Holt R. A., Goldowitz D., Hauswirth W. W., Wasserman W. W., Simpson E. M. (2014) “Targeted CNS Delivery Using Human MiniPromoters and Demonstrated Compatibility with Adeno-Associated Viral Vectors.” Mol Ther Methods Clin Dev 1: 5.
  • Dinculescu A., Min S.-H., Deng W.-T., Li Q., Hauswirth W. W. (2014) Gene Therapy in the Rd6 Mouse Model of Retinal Degeneration. Retinal Degenerative Diseases. J. D. Ash, C. Grimm, J. G. Hollyfield et al., Springer New York. 801: 711-718.
  • Dyka F. M., Boye S. L., Chiodo V. A., Hauswirth W. W., Boye S. E. (2014) “Dual Adeno-Associated Virus Vectors Result in Efficient In Vitro and In Vivo Expression of an Oversized Gene, MYO7A.” Human Gene Therapy Methods 25: 166-177.
  • Dyka F. M., Boye S. L., Ryals R. C., Chiodo V. A., Boye S. E., Hauswirth W. W. (2014) Cone Specific Promoter for Use in Gene Therapy of Retinal Degenerative Diseases. Retinal Degenerative Diseases. J. D. Ash, C. Grimm, J. G. Hollyfield et al., Springer New York. 801: 695-701.
  • Ezra-Elia R., Banin E., Honig H., Rosov A., Obolensky A., Averbukh E., Hauswirth W., Gootwine E., Ofri R. (2014) “Flicker cone function in normal and day blind sheep: a large animal model for human achromatopsia caused by CNGA3 mutation.” Documenta Ophthalmologica: 1-10.
  • Fu S., Zhu M., Ash J. D., Wang Y., Le Y.-Z. (2014) Investigating the Role of Retinal Müller Cells with Approaches in Genetics and Cell Biology. Retinal Degenerative Diseases. J. D. Ash, C. Grimm, J. G. Hollyfield et al., Springer New York. 801: 401-405.
  • Furlanetto R. L., De Moraes C. G., Teng C. C., Liebmann J. M., Greenfield D. S., Gardiner S. K., Ritch R., Krupin T. (2014) “Risk Factors for Optic Disc Hemorrhage in the Low-Pressure Glaucoma Treatment Study.” American Journal of Ophthalmology 157: 945-952.e941.
  • Giordano B. P., Tuli S. Y., Tuli S. S. (2014) “An Uncommon Cause for a Preschool Child’s Chronic Cough.” Journal of Pediatric Health Care 28: 267-271.
  • Gonzalez A., Fakhar K., Gubernick D., Tuli S. (2014) “Scleritis Caused by In Vitro Linezolid-Resistant Nocardia asteroides.” Case Reports in Ophthalmological Medicine 2014: 3.
  • Ha J.-H., Shil P. K., Zhu P., Gu L., Li Q., Chung S. (2014) “Ocular Inflammation and Endoplasmic Reticulum Stress Are Attenuated by Supplementation with Grape Polyphenols in Human Retinal Pigmented Epithelium Cells and in C57BL/6 Mice.” The Journal of Nutrition 144: 799-806.
  • Hauswirth W. W. (2014) “Retinal Gene Therapy Using Adeno-Associated Viral Vectors: Multiple Applications for a Small Virus.” Human Gene Therapy 25: 671-678.
  • Kelly M. N., Tuli S. Y., Tuli S. S., Stern M. A., Giordano B. P. “Brothers with Smith-Lemli-Opitz Syndrome.” Journal of Pediatric Health Care.
  • Kim Y., Tarallo V., Kerur N., Yasuma T., Gelfand B. D., Bastos-Carvalho A., Hirano Y., Yasuma R., Mizutani T., Fowler B. J., Li S., Kaneko H., Bogdanovich S., Ambati B. K., Hinton D. R., Hauswirth W. W., Hakem R., Wright C., Ambati J. (2014) “DICER1/Alu RNA dysmetabolism induces Caspase-8–mediated cell death in age-related macular degeneration.” Proceedings of the National Academy of Sciences.
  • Kohli N., Westerveld D. R., Ayache A. C., Verma A., Shil P., Prasad T., Zhu P., Chan S. L., Li Q., Daniell H. (2014) “Oral Delivery of Bioencapsulated Proteins Across Blood-Brain and Blood-Retinal Barriers.” Mol Ther 22: 535-546.
  • Koilkonda R. D., Yu H., Chou T. H., Feuer W. J., Ruggeri M., Porciatti V., Tse D., Hauswirth W. W., Chiodo V., Boye S. L., Lewin A. S., Neuringer M., Renner L., Guy J. (2014) “Safety and effects of the vector for the leber hereditary optic neuropathy gene therapy clinical trial.” JAMA Ophthalmology 132: 409-420.
  • Koilkonda R. D., Yu H., Talla V., Porciatti V., Feurer W. J., Hauswirth W. W., Chiodo V. A., Erger K., Boye S. L., Lewin A. S., Conlon T. J., Renner L., Neuringer M., Detrisac C., Guy J. (2014) “LHON Gene Therapy Vector Prevents Visual Loss and Optic Neuropathy Induced by G11778A Mutant Mitochondrial DNA: Biodistribution and Toxicology Profile.” Investigative Ophthalmology & Visual Science.
  • Ku C. A., Chiodo V. A., Boye S. L., Hayes A., Goldberg A. F. X., Hauswirth W. W., Ramamurthy V. (2014) “Viral-mediated vision rescue of a novel AIPL1 cone-rod dystrophy model.” Human Molecular Genetics.
  • Marchette L. D., Sherry D. M., Brush R. S., Chan M., Wen Y., Wang J., Ash J. D., Anderson R. E., Mandal N. A. (2014) Very Long Chain Polyunsaturated Fatty Acids and Rod Cell Structure and Function. Retinal Degenerative Diseases. J. D. Ash, C. Grimm, J. G. Hollyfield et al., Springer New York. 801: 637-645.
  • McClintock M., Peden M. C., Kay C. N. (2014) Spectral Domain Optical Coherence Tomography Findings in CNGB3-Associated Achromatopsia and Therapeutic Implications. Retinal Degenerative Diseases. J. D. Ash, C. Grimm, J. G. Hollyfield et al., Springer New York. 801: 551-557.
  • Mowat F. M., Gornik K. R., Dinculescu A., Boye S. L., Hauswirth W. W., Petersen-Jones S. M., Bartoe J. T. (2014) “Tyrosine capsid-mutant AAV vectors for gene delivery to the canine retina from a subretinal or intravitreal approach.” Gene Ther 21: 96-105.
  • Netland P. A., Sarkisian Jr S. R., Moster M. R., Ahmed I. I. K., Condon G., Salim S., Sherwood M. B., Siegfried C. J. (2014) “Randomized, Prospective, Comparative Trial of EX-PRESS Glaucoma Filtration Device versus Trabeculectomy (XVT Study).” American Journal of Ophthalmology 157: 433-440.e433.
  • O’Reilly M., Federoff H. J., Fong Y., Kohn D. B., Patterson A. P., Ahmed N., Asokan A., Boye S. E., Crystal R. G., De Oliveira S., Gargiulo L., Harper S. Q., Ikeda Y., Jambou R., Montgomery M., Prograis L., Rosenthal E., Sterman D. H., Vandenberghe L. H., Zoloth L., Abedi M., Adair J., Adusumilli P. S., Goins W. F., Gray J., Monahan P., Popplewell L., Sena-Esteves M., Tannous B., Weber T., Wierda W., Gopal-Srivastava R., McDonald C. L., Rosenblum D., Corrigan-Curay J. (2014) “Gene Therapy: Charting a Future Course—Summary of a National Institutes of Health Workshop, April 12, 2013.” Human Gene Therapy 25: 488-497.
  • Prasad T., Verma A., Li Q. (2014) “Expression and cellular localization of the Mas receptor in the adult and developing mouse retina.” Molecular Vision 20: 1443-1455.
  • Qiu Y., Shil P. K., Zhu P., Yang H., Verma A., Lei B., Li Q. (2014) “Angiotensin-Converting Enzyme 2 (ACE2) Activator Diminazene Aceturate Ameliorates Endotoxin-Induced Uveitis in Mice.” Investigative Ophthalmology & Visual Science 55: 3809-3818.
  • Ramamurthy V., Jolicoeur C., Koutroumbas D., Mühlhans J., Le Y.-Z., Hauswirth W. W., Giessl A., Cayouette M. (2014) “Numb Regulates the Polarized Delivery of Cyclic Nucleotide-Gated Ion Channels in Rod Photoreceptor Cilia.” The Journal of Neuroscience 34: 13976-13987.
  • Regus-Leidig H., Fuchs M., Löhner M., Leist S., Leal-Ortiz S., Chiodo V., Hauswirth W., Garner C., Brandstätter J. H. (2014) “In vivo knockdown of Piccolino disrupts presynaptic ribbon morphology in mouse photoreceptor synapses.” Frontiers in Cellular Neuroscience 8.
  • Shenoy V., Kwon K.-C., Rathinasabapathy A., Lin S., Jin G., Song C., Shil P., Nair A., Qi Y., Li Q., Francis J., Katovich M. J., Daniell H., Raizada M. K. (2014) “Oral Delivery of Angiotensin-Converting Enzyme 2 and Angiotensin-(1-7) Bioencapsulated in Plant Cells Attenuates Pulmonary Hypertension.” Hypertension.
  • Shifera A. S., Kay C. N. “Early-onset X-linked Retinitis Pigmentosa in a Heterozygous Female Harboring an Intronic Donor Splice Site Mutation in the Retinitis Pigmentosa GTPase Regulator Gene.” Ophthalmic Genetics 0: 1-6.
  • Shifera A. S., Leoncavallo A., Sherwood M. (2014) “Probable Association of an Attack of Bilateral Acute Angle-Closure Glaucoma With Duloxetine.” Annals of Pharmacotherapy 48: 936-939.
  • Shil P. K., Kwon K.-C., Zhu P., Verma A., Daniell H., Li Q. (2014) “Oral Delivery of ACE2/Ang-(1-7) Bioencapsulated in Plant Cells Protects against Experimental Uveitis and Autoimmune Uveoretinitis.” Mol Ther.
  • Sriram S., Gibson D. J., Robinson P., Pi L., Tuli S., Lewin A. S., Schultz G. (2014) “Assessment of anti-scarring therapies in ex vivo organ cultured rabbit corneas.” Experimental Eye Research 125: 173-182.
  • Talla V., Porciatti V., Chiodo V., Boye S. L., Hauswirth W. W., Guy J. (2014) “Gene Therapy With Mitochondrial Heat Shock Protein 70 Suppresses Visual Loss and Optic Atrophy in Experimental Autoimmune Encephalomyelitis.” Investigative Ophthalmology & Visual Science 55: 5214-5226.
  • Tuli S., Kelly M., Ryan K., Tuli S., Giordano B. P. (2014) “A 4-Year-Old Child Who Could Not Supinate Her Forearm.” Journal of Pediatric Health Care 28: 357-360.
  • Wilson A. M., Chiodo V. A., Boye S. L., Brecha N. C., Hauswirth W. W., Di Polo A. (2014) “Inhibitor of Apoptosis-Stimulating Protein of p53 (iASPP) Is Required for Neuronal Survival after Axonal Injury.” PLoS ONE 9: e94175.
  • Yang H., Zheng S., Qiu Y., Yang Y., Wang C., Yang P., Li Q., Lei B. (2014) “Activation of Liver X Receptor Alleviates Ocular Inflammation in Experimental Autoimmune Uveitis.” Investigative Ophthalmology & Visual Science 55: 2795-2804.
  • Zhu Y., Xu J., Hauswirth W. W., DeVries S. H. (2014) “Genetically Targeted Binary Labeling of Retinal Neurons.” The Journal of Neuroscience 34: 7845-7861.

 

2013 Center Research

  • Christine N. Kay,  Renee C. Ryals, , George V. Aslanidi, , Seok Hong Min,  Qing Ruan, Jingfen Sun, Frank M. Dyka, Daniel Kasuga, Andrea E. Ayala, Kim Van Vliet, Mavis Agbandje-McKenna, William W. Hauswirth, Sanford L. Boye, and Shannon E. Boye. Targeting photoreceptors via intravitreal delivery using novel, capsid-mutated AAV vectors. PLoS One (in press).
  • Thomas J. Conlon, Wen-Tao Deng, Kristen Erger, Travis Cossette, Ji-jing Pang, Renee Ryals, Nathalie Clement, Brian Cleaver, Issam McDoom, Shannon E. Boye, Marc C. Peden, Mark B. Sherwood, Corrine R. Abernathy, Fowzan S. Alkuraya, Sanford L. Boye, William W. Hauswirth. Preclinical potency and safety studies of an AAV2-mediated gene therapy vector for the treatment of MERTK associated retinitis pigmentosa. Human Gene Therapy (in press).
  • Li Jiang, Tansy Z. Li, Shannon E. Boye, William W. Hauswirth, Jeanne M. Frederick, Wolfgang Baehr. RNAi-mediated gene suppression in a GCAP1(L151F) cone-rod mouse model. PLoS One. 2013;8(3):e57676. doi: 10.1371/journal.pone.0057676. Epub 2013 Mar 5
  • Vanda Lopes*, Shannon E. Boye*, Carrie M. Louie, Sanford Boye, Frank Dyka, Vince Chiodo, William W. Hauswirth, D. S. Williams.  “Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus”. Gene Therapy  2012 Jan 24. doi: 10.1038/gt.2013.3. [Epub ahead of print] *equal contribution
  • Shannon E. Boye, Sanford L. Boye, Alfred Lewin, William W. Hauswirth. “A Comprehensive Review of Retinal Gene Therapy”. Molecular Therapy 2013 Mar;21(3):509-19. doi: 10.1038/mt.2012.280. Epub 2013 Jan 29.
  • Sanford L. Boye,   Igor V. Peshenko,  Wei Chieh Huang,  Seok Hong Min, Issam McDoom, Xuan Liu, Frank M. Dyka, Tom C. Foster, Yumiko Umino, Sukanya Karan, Samuel G. Jacobson,Wolfgang Baehr, Alexander Dizhoor, William W. Hauswirth and Shannon E. Boye. “AAV-mediated gene therapy in the guanylate cyclase (RetGC1/RetGC2) double knockout mouse model of Leber congenital amaurosis”. Human Gene Therapy 2013 Feb;24(2):189-202. doi: 10.1089/hum.2012.193
  • Bhalla S, Joshi D, Bhullar S, Kasuga D, Park Y, Kay CN. Long term follow-up data for efficacy and safety of treatment of retinitis pigmentosa with valproic acid.” Accepted to British Journal Ophthalmology.
  • Hollsten JE, McClintock M, Samy H, Peden M, Kay CN. “Fulminant chorioretinitis and papillitis secondary to coxsackievirus B virus presenting as acute posterior multifocal placoid pigment epitheliopathy with positive response to IVIG.” Accepted to Retina Cases and Brief Reports.
  • McClintock M, Peden MC, Kay CN. “Spectral domain optical coherence tomography findings in siblings with CNGB3-associated achromatopsia.” Accepted to Adv. Exp. Med. Biol.
  • Ye Tao, Tao Chen, Bei Liu, Jun Hui Xue, Lei Zhang, Feng Xia, Ji-jing Pang and Zuo Ming Zhang. Visual signal pathway reorganization in the cacna1f mutant rat model. Invest Ophthalmol Vis Sci. 2013; 54(3): 1988-97. doi: 10.1167/iovs.12-10706.
  • Xufeng Dai and Ji-Jing Pang. Progress on study of achromatopsia and targeted gene therapy. Chinese Journal of Ophthalmology. 2012; 8: 755-758.
  • Battelle, B-A. What the clock tells the eye: Lessons from an ancient arthropod. Integrative and Comparative. Biology. (accepted for publication)
  • Battelle, B-A., Kempler, K.E., Parker, A.K., Gaddie C.D. Opsin1-2, Gqα and arrestin levels at Limulus rhabdoms are controlled by diurnal light and a circadian    clock. J. Exp. Biol. 2013 Feb 7. [Epub ahead of  print]
  • Qi Y, Li Q, Shenoy V, Zingler M, Jun J, Verma A, Katovich MJ, Raizada MK. Comparison of the transduction efficiency of tyrosine-mutant adeno-associated virus serotype vectors in kidney. Clin Exp Pharmacol Physiol. 2013 Jan;40(1):53-5. doi: 10.1111/1440-1681.12037. PMID:23216315
  • J. Zubcevic, J. Jun, G. Lamont, T. M. Murça, P.Shi, W. Yuan, Q. Li, C. Sumners, M. K. Raizada, and Z. Shan.  NTS (pro)renin receptor-mediated antihypertensive effect involves NF-КBcytokine signaling in the spontaneously hypertensive rat (SHR). Hypertension, 2013 Mar; 61(3):622-7. doi:10.1161/ HYPERTENSIONAHA. 111.199836. Epub 2013 Jan 14. PMID:23319541
  • S.S. Radhakrishnan, P.M. Robinson, T.D. Blalock, G. Secker, J. Daniels, G.R. Grotendorst, G.S. Schultz.  Effect of Connective Tissue Growth Factor on Protein Kinase Expression and Activity in Human Corneal Fibroblasts. Invest Ophthalmol Sci, 53:8076-8085, 2013.

2012 Center Research

  • Jacobson SG, Cideciyan AV, Ratnakaram R, Heon E, Schwartz SB, Roman AJ, Peden MC, Aleman TS, Boye SL, Sumaroka A, Conlon TJ, Calcedo R, Pang JJ, Erger KE, Olivares MB, Mullins CL, Swider M, Kaushal S, Feuer WJ, Iannaccone A, Fishman GA, Stone EM, Byrne BJ, Hauswirth WW. Gene therapy for Leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years. Arch Ophthalmol. 2012; 130(1):9-24
  • Kay CN, Abramoff MD, Mullins RF, Kinnick TR, Lee K, Eyestone ME, Chung MM, Sohn EH, Stone EM. Three-dimensional distribution of the vitelliform lesion, photoreceptors, and retinal pigment epithelium in the macula of patients with best vitelliform macular dystrophy. Arch Ophthalmol. 2012; 130(3):357-64 .
  • Kay CN, Pavan PR, Small LB, Zhang T, Zamba GKD, Cohen SM. “Familial Trends in a Population with Macular Holes.”  Retina. 2012 Apr;32(4):754-9.
  • Russell SR, Sohn EH, Boldt HC, Folk JC, Tarantola RM, Kay CN, Mahajan VM. “Elimination of infusion bubbles and uncontrolled reflux in the Alcon constellation vitrectomy vision system.”  Retina. 2012 Dec 5. [Epub ahead of print]
  • Kay CN, Quiram P, Mahajan VM. “23-Gauge Pediatric Vitrectomy using Limbus-based Trocar-Cannulas.” Retina. 2012 May;32(5):1023-7.
  • Shannon E. Boye, John J. Alexander, Sanford L. Boye, Clark D. Witherspoon, Kristen J. Sandefer, Thomas J. Conlon, Kirsten Erger, Vince A. Chiodo, Mark E. Clark, Christopher A. Girkin, William W. Hauswirth, Paul D. Gamlin. The human rhodopsin kinase promoter in an AAV5 vector confers rod and cone specific expression in the primate retina. Human Gene Therapy 2012 Oct;23(10):1101-15.
  • Kunte MM, Choudhury S, Manheim JF, Shinde VM, Miura M, Chiodo VA, Hauswirth WW, Gorbatyuk OS, Gorbatyuk MS. ER stress is involved in T17M rhodopsin-induced retinal degeneration. Invest Ophthalmol Vis Sci. 2012; 53(7):3792-800
  • Deng W-T, Dinculescu A, Li Q, Boye SL, Li J, Gorbatyuk MS, Pang J, Chiodo VA, Matthes MT, Yasumura D, Liu L, Alkuraya FS, Zhang K, Vollrath D, LaVail MM, Hauswirth WW. Tyrosine-mutant AAV8 delivery of human MERTK provides long-term retinal preservation in RCS rats. Invest Ophthalmol Vis Sci. 2012; 53(4):1895-904
  • Qi X, Cai J, Ruan Q, Liu L, Boye SL, Chen Z, Hauswirth WW, Ryals RC, Shaw L, Caballero S, Grant MB, Boulton ME. γ-Secretase inhibition of murine choroidal neovascularization is associated with reduction of superoxide and proinflammatory cytokines. Invest Ophthalmol Vis Sci. 2012; 53(2):574-85
  • Blalock TD, Gibson DJ, Duncan MR, Tuli SS, Grotendorst GR, Schultz GS. A connective tissue growth factor signaling receptor in corneal fibroblasts. Invest Ophthalmol Vis Sci. 2012; 53(7):3387-94
  • Min J, Lukowski ZL, Levine MA, Meyers CA, Beattie AR, Schultz GS, Samuelson DA, Sherwood MB. Comparison of single versus multiple injections of the protein saratin for prolonging bleb survival in a rabbit model. Invest Ophthalmol Vis Sci. 2012; 53(12):7625-30
  • Lee AG, Oetting TA, Blomquist PH, Bradford G, Culican SM, Kloek C, Krishnan C, Lauer AK, Levi L, Naseri A, Rubin SE, Scott IU, Tao J, Tuli S, Wright MM, WuDunn D, Zimmerman MB. A multicenter analysis of the ophthalmic knowledge assessment program and American Board of Ophthalmology written qualifying examination performance. Ophthalmology. 2012; 119(10):1949-53
  • Beltran WA, Cideciyan AV, Lewin AS, Iwabe S, Khanna H, Sumaroka A, Chiodo VA, Fajardo DS, Roman AJ, Deng W-T, Swider M, Aleman TS, Boye SL, Genini S, Swaroop A, Hauswirth WW, Jacobson SG, Aguirre GD. Gene therapy rescues photoreceptor blindness in dogs and paves the way for treating human X-linked retinitis pigmentosa. Proc Nat Acad Sci. 2012; 109(6):2132-7
  • Bermingham-McDonogh O, Corwin JT, Hauswirth WW, Heller S, Reed R, Reh TA. Regenerative medicine for the special senses: restoring the inputs. J Neurosci. 2012; 32(41):14053-7
  • Chollangi S, Mather T, Rodgers KK, Ash JD. A unique loop structure in oncostatin M determines binding affinity toward oncostatin M receptor and Leukemia Inhibitory Factor receptor. J Biol Chem. 2012; 287(39):32848-59
  • Chucair-Elliott AJ, Elliott MH, Wang J, Moiseyev GP, Ma J-X, Politi LE, Rotstein NP, Akira S, Uematsu S, Ash JD. Leukemia Inhibitory Factor coordinates the down-regulation of the visual cycle in the retina and retinal-pigmented epithelium. J Biol Chem. 2012; 287(29):24092-102
  • Dinculescu A, Estreicher J, Zenteno JC, Aleman TS, Schwartz SB, Huang WC, Roman AJ, Sumaroka A, Li Q, Deng WT, Min SH, Chiodo VA, Neeley A, Liu X, Shu X, Matias-Florentino M, Buentello-Volante B, Boye SL, Cideciyan AV, Hauswirth WW, Jacobson SG. Gene therapy for retinitis pigmentosa caused by MFRP mutations: human phenotype and preliminary proof of concept. Hum Gene Ther. 2012; 23(4):367-76
  • Doroudchi MM, Greenberg KP, Zorzos AN, Hauswirth WW, Fonstad CG, Horsager A, Boyden ES. Towards optogenetic sensory replacement. Proc IEEE Eng Med Biol Soc. 2012; 2011:3139-41 (Univ. Florida — Unres. Grant). Keyword(s): Artificial Vision; Gene Research/Therapy
  • Dridi S, Hirano Y, Tarallo V, Kim Y, Fowler BJ, Ambati BK, Bogdanovich S, Chiodo VA, Hauswirth WW, Kugel JF, Goodrich JA, Ponicsan SL, Hinton DR, Kleinman ME, Baffi JZ, Gelfand BD, Ambati J. ERK1/2 activation is a therapeutic target in age-related macular degeneration. Proc Nat Acad Sci. 2012; 109(34):13781-6
  • Gedde SJ, Singh K, Schiffman JC, Feuer WJ, Group TVTS. The tube versus trabeculectomy study: interpretation of results and application to clinical practice. Curr Opin Ophthalmol. 2012; 23(2):118-26
  • Gorbatyuk MS, Gorbatyuk OS, LaVail MM, Lin JH, Hauswirth WW, Lewin AS, LaVail MM, Ash JD, Anderson RE, Hollyfield JG, Grimm C. Functional rescue of P23H rhodopsin photoreceptors by gene delivery retinal degenerative diseases. Adv Exp Med Biol. 2012; 723:191-7
  • D.J. Gibson and G.S. Schultz. Ectopic Epithelial Implants Following Surface Ablation Of The Cornea. Invest Ophthalmol Vis Sci, 53:7760-7765, 2012.
  • Kelly MN, Khuddus N, Motamarry S, Tuli S. Microcephaly, lymphedema, chorioretinal dysplasia (MLCRD) syndrome. J Ped Health Care. 2012; 26(4):306-11
  • Knape RM, Motamarry SP, Clark CL, Bohsali KI, Khuddus N. Morning glory disc anomaly and optic nerve coloboma. Clinical Pediatrics. 2012; 51(10):991-3
  • Li X, McClellan ME, Tanito M, Garteiser P, Towner R, Bissig D, Berkowitz BA, Fliesler SJ, Woodruff ML, Fain GL, Birch DG, Khan MS, Ash JD, Elliott MH. Loss of caveolin-1 impairs retinal function due to disturbance of subretinal microenvironment. J Biol Chem. 2012; 287(20):16424-34
  • Light AC, Zhu Y, Shi J, Saszik S, Lindstrom S, Davidson L, Li X, Chiodo VA, Hauswirth WW, Li W, DeVries SH. Organizational motifs for ground squirrel cone bipolar cells. J Comp Neurol. 2012; 520(13):2864-87
  • Mao H, Gorbatyuk MS, Hauswirth WW, Lewin AS, LaVail MM, Ash JD, Anderson RE, Hollyfield JG, Grimm C. Gene delivery of wild-type rhodopsin rescues retinal function in an autosomal dominant retinitis pigmentosa mouse model. Adv Exp Med Biol. 2012; 723:199-205
  • Mao H, Gorbatyuk MS, Rossmiller B, Hauswirth WW, Lewin AS. Long-term rescue of retinal structure and function by rhodopsin RNA replacement with a single adeno-associated viral vector in P23H RHO transgenic mice. Hum Gene Ther. 2012; 23:356-66
  • Min J, Lukowski ZL, Levine MA, Meyers CA, Beattie AR, Schultz GS, Samuelson DA, Sherwood MB. Prevention of ocular scarring post glaucoma filtration surgery using the inflammatory cell and platelet binding modulator saratin in a rabbit model. PLoS One. 2012; 7(4):e35627
  • R.R. Mohan, J.C.K. Tovey, A. Sharma, G.S. Schultz, J.W. Cowden, A. Tandon.  Targeted Decorin Gene Therapy Delivered with Adeno-Associated Virus Effectively Retards Corneal Neovascularization In Vivo.  PLoS ONE, 6: e26432, 2012.
  • Ohlstein DH, Hooten C, Perez J, Clark CL, Samy H. Orbital aspergillosis:  voriconazole, the new standard in treatment. Case Rep Ophthalmol. 2012; 3(1):46-53
  • Pang J-J, Deng W-T, Dai X, Lei B, Everhart D, Umino Y, Li J, Zhang K, Mao S, Boye SL, Liu L, Chiodo VA, Liu X, Shi W, Tao Y, Chang B, Hauswirth WW. AAV-mediated cone rescue in a naturally occurring mouse model of CNGA3-achromatopsia. PLoS One. 2012; 7(4):e35250
  • Petrs-Silva H, Yasumura D, Matthes MT, LaVail MM, Lewin AS, Hauswirth WW, LaVail MM, Ash JD, Anderson RE, Hollyfield JG, Grimm C. Suppression of rds expression by siRNA and gene replacement strategies for gene therapy using rAAV vector. Adv Exp Med Biol. 2012; 723:215-23
  • Pi L, Shenoy AK, Liu J, Kim S, Nelson N, Xia H, Hauswirth WW, Petersen BE, Schultz GS, Scott EW. CCN2/CTGF regulates neovessel formation via targeting structurally conserved cystine knot motifs in multiple angiogenic regulators. FASEB J. 2012; 26(8):3365-7
  • P.M. Robinson, T.S. Smith, D. Patel, M. Dave, A.S. Lewin, L. Pi, E.W. Scott,  S.S. Tuli, G.S. Schultz.  Proteolytic Processing of Connective Tissue Growth Factor in Normal Ocular Tissues and During Corneal Wound Healing. Invest Ophthalmol Vis Sci. 53:8093-8103, 2012.
  • P.M. Robinson, T.D. Blalock, R. Yuan, A.S. Lewin, G.S. Schultz. Hammerhead ribozyme-mediated knockdown of mRNA for fibrotic growth factors: transforming growth factor-beta 1 and connective tissue growth factor. Methods Mol Biol, 820: 117-132, 2012
  • Seibold LK, Sherwood MB, Kahook MY. Wound modulation after filtration surgery. Survey Ophthalmol. 2012; 57(6):530-50
  • Tarallo V, Hirano Y, Gelfand B, Dridi S, Kerur N, Kim Y, Cho Won G, Kaneko H, Fowler Benjamin J, Bogdanovich S, Albuquerque R, Hauswirth William W, Chiodo Vince A, Kugel Jennifer F, Goodrich James A, Ponicsan Steven L, Chaudhuri G, Murphy Michael P, Dunaief Joshua L, Ambati Balamurali K, Ogura Y, Yoo Jae W, Lee D-k, Provost P, Hinton David R, Nunez G, Baffi Judit Z, Kleinman Mark E, Ambati J. DICER1 loss and Alu RNA induce age-related macular degeneration via the NLRP3 inflammasome and MyD88. Cell. 2012; 149(4):847-59
  • A. Tandon, J.C. Tovey, M.R. Waggonor, A. Sharma, J.W. Cowden, V. Lopez, Y. Liu, D.J. Gibson; G.S. Schultz, R.R. Mohan. Vorinostat: A Potent Agent to Prevent Laser-Induced Haze in the Cornea In Vivo.  J Refractive Surgery, 28:285-290, 2012.
  • Thampi P, Rao HV, Mitter SK, Cai J, Mao H, Li H, Seo S, Qi X, Lewin AS, Romano C, Boulton ME. The 5HT1a receptor agonist 8-OH DPAT induces protection from lipofuscin accumulation and oidative Stress in the retinal pigment epithelium. PLoS One. 2012; 7(4):e34468 (Univ. Florida — Unres. Grant). RPB support has been inadvertently omitted by grantee institution. Keyword(s): Retina/Retinal Diseases
  • Tuli S, Tuli S. A 6-year-old girl with restricted upward gaze of her right eye. Pediatr Rev. 2012; 33(8):e53-6
  • Tuli SY, Kelly M, Giordano B, Fillipps DJ, Tuli SS. Blepharoptosis: assessment and management. J Ped Health Care. 2012; 26(2):149-54
  • Tuo J, Pang J-J, Cao X, Shen D, Zhang J, Scaria A, Wadsworth SC, Pechan P, Boye SL, Hauswirth WW, Chan C-C. AAV5-mediated sFLT01 gene therapy arrests retinal lesions in Ccl2(-/-)/Cx3cr1(-/-) mice. Neurobiol Aging. 2012; 33(2):433.e1-10
  • Verma A SZ, Lei B, Yuan L, Liu X, Nakagawa T, Grant MB, Lewin AS, Hauswirth WW, Raizada MK, Li Q. ACE2 and Ang-(1-7) confer protection against development of diabetic retinopathy. Mol Ther. 2012; 20:28-36
  • Wang H MR, Taaffe D, Yin S, Xia L, Hauswirth WW, Bance M, Robertson GS, Wang J.Efficient cochlear gene transfection in guinea-pigs with adeno-associated viral vectors by partial digestion of round window membrane. Gene Ther. 2012; 19(3):255-63
  • Yao J, Jia L, Khan N, Zheng Q-D, Moncrief A, Hauswirth WW, Thompson DA, Zacks DN. Caspase inhibition with XIAP as an adjunct to AAV vector gene-replacement therapy: improving efficacy and prolonging the treatment window. PLoS One. 2012; 7(5):e37197
  • Yellowlees Douglas J, Bhatwadekar AD, Li Calzi S, Shaw LC, Carnegie D, Caballero S, Li Q, Stitt AW, Raizada MK, Grant MB. Bone marrow-CNS connections: Implications in the pathogenesis of diabetic retinopathy. Prog Ret Eye Res. 2012; 31(5):481-94
  • Yu H, Koilkonda RD, Chou T-H, Porciatti V, Ozdemir SS, Chiodo V, Boye SL, Boye SE, Hauswirth WW, Lewin AS, Guy J. Gene delivery to mitochondria by targeting modified adenoassociated virus suppresses Leber’s hereditary optic neuropathy in a mouse model. Proc Nat Acad Sci. 2012; 109(20):E1238-47
  • Yu H, Ozdemir SS, Koilkonda RD, Chou TH, Porciatti V, Chiodo V, Boye SL, Hauswirth WW, Lewin AS, Guy J. Mutant NADH dehydrogenase subunit 4 gene delivery to mitochondria by targeting sequence-modified adeno-associated virus induces visual loss and optic atrophy in mice. Mol Vis. 2012; 18:1668-83
  • Qinxiang Zheng, Yueping Ren1, Radouil Tzekov, Yuanping Zhang, Bo Chen, Jiangping Hou, Chunhui Zhao, Jiali Zhu, Ying Zhang, Xufeng Dai, Shan Ma, Jia Li, Ji-jing Pang, Jia Qu, Wensheng Li. Differential proteomics and functional research following gene therapy in a mouse model of Leber congenital amaurosis. PLoS ONE. 2012; 7(8):e44855. Epub 2012 Aug 31
  • Qinxiang Zheng, Shelley Yang, Yuanping Zhang,Ronghan Wu, Ji-jing Pang, Wensheng Li. Vitreous surgery for macular hole-related retinal detachment after phacoemulsification cataract extraction: Ten-year retrospective review. Eye (2012) 26, 1058–1064.
  • Hazra S, Rasheed A, Bhatwadekar A, Wang X, Shaw LC, Patel M, Caballero S, Magomedova L, Solis N, Yan Y, Wang W, Thinschmidt JS, Verma A,Li Q, Levi M, Cummins CL, Grant MB.  Liver X Receptor Modulates Diabetic Retinopathy Outcome in a Mouse Model of Type 1 Diabetes. Diabetes. 2012 Dec;61(12):3270-9. doi: 10.2337/db11-1596.  Epub 2012 Aug 13. PMID:22891211
  • Z.l. Lukowski, J. Min, A.R. Beattie, C.A. Meyers, M.A. Levine, G.S. Schultz, D.A. Samuelson, M.B. Sherwood.  Prevention of Ocular Scarring Following Glaucoma Surgery using the Monoclonal Antibody LT1009 (Sonepcizumab™) in a Rabbit Model, J Glaucoma, in press

2011 Center Research

  • Jiang L, Zhang H, Dizhoor AM, Boye SE, Hauswirth WW, Frederick JM, Baehr W. Long term interference gene therapy in a dominant retinitis pigmentosa mouse model. Proc Natl Acad Sci U S A (2011) Nov 8;108(45):18476-81.
  • Sanford L. Boye, Thomas Conlon, Kirsten Erger, Renee Ryals, Andy Neeley, Travis Cossette, Jijing Pang, Frank M. Dyka, William W. Hauswirth, Shannon E. Boye. (2011) Long term preservation of cone photoreceptors and restoration of cone function by gene therapy in the guanylate cyclase-1 knockout (GC1KO) mouse. Invest Ophthalmol Vis Sci 52:7098-108. PMID: 21778276 http://www.ncbi.nlm.nih.gov/pubmed/21778276
  • Cai J, Chen Z, Ruan Q, Han S, Liu L, Qi X, Boye SL, Hauswirth WW, Grant MB, Boulton ME. g-Secretase and Presenilin Mediate Cleavage and Phosphorylation of Vascular Endothelial Growth Factor Receptor-1. J Biol Chem 2011; In press.
  • Cai J, Han S, Qing R, Liao D, Law B, Boulton ME.  (2011)  In pursuit of new anti-angiogenic therapies for cancer treatment.  Front Biosci. 16:803-14.
  • Cai J, Wu L, Qi X, Shaw L, Li Calzi S, Caballero S, Jiang WG, Vinores SA, Antonetti D, Ahmed A, Grant MB, Boulton ME.  (2011)  Placenta Growth Factor-1 Exerts Time-Dependent Stabilization of Adherens Junctions Following VEGF-Induced Vascular Permeability.  PLoS One. 2011 Mar 25;6(3):e18076.
  • Cai J, Wu L, Qi X, Li Calzi S, Caballero S, Shaw L, Ruan Q, Grant MB, Boulton ME.  (2011)  PEDF regulates vascular permeability by a γ-secretase-mediated pathway.   PLoS One. 2011;6(6):e21164. Epub 2011 Jun 17
  • Chan, F, Hauswirth, WW,  Wensel, TG and Wilson, JH “Efficient Mutagenesis of the Rhodopsin Gene in Rod Photoreceptor Neurons in Mice” Nucl. Acids Res.  In Press (2011) Apr 7. [Epub ahead of print] PMID:21478169
  • Ku CA, Chiodo VA, Boye SL, Goldberg AF, Li T, Hauswirth WW, Ramamurthy V. “Gene therapy using self-complementary Y733F capsid mutant AAV2/8 restores vision in a model of early onset Leber congenital amaurosis.”Hum Mol Genet. 2011 Aug 31. [Epub ahead of print] PMID:21880665
  • M. Mehdi Doroudchi, Kenneth P. Greenberg, Jianwen Liu, Kimberly A. Silka, Edward S. Boyden, Jennifer A. Lockridge, A. Cyrus Arman, Ramesh Janani, Shannon E. Boye, Sanford L. Boye, Gabriel M. Gordon, Benjamin C. Matteo, Alapakkam P. Sampath, William W. Hauswirth, Alan Horsager. (2011) Virally-Delivered Channelrhodopsin-2 Safely and Effectively Restores Visual Function in Multiple Mouse Models of Blindness. Mol Ther. 19:1220-9 PMID: 21505421 http://www.ncbi.nlm.nih.gov/pubmed/21505421
  • Esmaeelpour M, Watts PO, Boulton ME, Cai J, Murphy PJ. (2011) Tear Film Volume and Protein Analysis in Full-term Newborn Infants. Cornea. Oct 28. 30:400-404.
  •  D.J. Gibson and G.S. Schultz.  A Corneal Scarring Model.  In: Wound Regeneration and Repair: Methods and Protocols, R. Gourdie, editor, Humana Press, Totowa New Jersey, in press.
  • Jacobson, SG, Cideciyan, AV, Ratnakaram, Heon, E, Schwartz, SB, Roman, AJ, Peden, MC, Aleman, TS,  Boye, SL, Sumaroka, A, Conlon, TJ, Calcedo, R, Pang, J, Erger, KE, Olivares, MB, Mullins, CL,Swider, M, Kaushal, S, Feuer, WJ, Iannaccone, A, Fishman, GA, Stone, EM, Byrne, BJ and Hauswirth, WW “Gene Therapy for Leber Congenital Amaurosis caused by RPE65 mutations: Safety and Efficacy in Fifteen Children and Adults Followed up to Three Years” Arch. Ophthal.  In Press (2011).
  • Ji-jing Pang, Lei Lei, Xufeng Dai, Wei Shi, Xuan Liu, Astra Dinculescu, J. Hugh McDowell. AAV-mediated gene therapy in mouse models of recessive retinal degeneration. Current Molecular Medicine; 2011 (In press).
  •  Kaneko H, Dridi S, Tarallo V, Gelfand BD, Fowler BJ, Cho WG, Kleinman ME, Ponicsan SL, Hauswirth WW, Chiodo VA, Karikó K, Yoo JW, Lee DK, Hadziahmetovic M, Song Y, Misra S, Chaudhuri G, Buaas FW, Braun RE, Hinton DR, Zhang Q, Grossniklaus HE, Provis JM, Madigan MC, Milam AH, Justice NL, Albuquerque RJ, Blandford AD, Bogdanovich S, Hirano Y, Witta J, Fuchs E, Littman DR, Ambati BK, Rudin CM, Chong MM, Provost P, Kugel JF, Goodrich JA, Dunaief JL, Baffi JZ, Ambati J. “DICER1 deficit induces Alu RNA toxicity in age-related macular degeneration.” Nature. 2011 Feb 6. [Epub ahead of print] PMID: 21297615
  • Kielczewski JL, Calzi SL, Shaw LC, Cai J, Qi X, Ruan Q, Wu L, Liu L, Hu P, Chan-Ling T, Mames RN, Firth S, Baxter RC, Turowski P, Busik JV, Boulton ME, Grant MB.  (2011)  Free Insulin-like Growth Factor Binding Protein-3 (IGFBP-3) reduces retinal vascular permeability in association with a reduction of acid sphingomyelinase (ASMase).  Invest Ophthalmol Vis Sci  52(11):8278-86.
  • Lei Y, Garrahan N, Hermann B, Fautsch MP, Johnson DH, Hernandez MR, Boulton M, Morgan JE. (2011) Transretinal degeneration in ageing human retina: a multiphoton microscopy analysis. Br J Ophthalmol. 2010 95;727-730.
  • Li Jiang, Houbin Zhang, Alexander Dizhoor, Shannon E. Boye, William W. Hauswirth, Jeanne Frederick, Wolfgang Baehr. RNA interference-based gene therapy in a dominant retinitis pigmentosa mouse model. (2011) Proc Natl Acad Sci U S A. In press http://www.ncbi.nlm.nih.gov/pubmed/22042849
  • Lin H, Xu H, Liang F-Q, Hao L, Gupta P, Havey AN, Boulton ME, Godley BF.  (2011)  Mitochondrial DNA damage and repair in RPE associated with aging and age-related macular degeneration.  Invest Ophthalmol Vis Sci 52(6):3521-9.
  •  Li X, Li W, Dai X, Kong F, Zeng Q, Zhou X, Lü F, Chang B, Rohrer B, Hauswirth WW, Qu J, Pang JJ.”Gene Therapy Rescues Cone Structure and Function in the Three-Month-Old rd12 Mouse: A Model for Mid-Course RPE65 Leber Congenital Amaurosis.”Invest Ophthalmol Vis Sci. 52:7-15 2010 PMID: 21169527
  • Ma A, Zhao B, Boulton M, Albon J.(2011)  A role for Notch signaling in corneal wound healing. Wound Repair Regen. 19:98–106
  • Mao, H, Thomas, Jr., J, Schwein, A, Shabashvili, AE, Hauswirth, WW, Gorbatyuk, MS, Lewin, AS, AAV Delivery of Wild-Type Rhodopsin Preserves Retinal Function in a Mouse Model of Autosomal Dominant Retinitis Pigmentosa, Hum. Gene Ther. 5:567-575 (2011).
  • Mi, S., Dooley, E.P., Albon, J., Boulton, M.E., Meek, K.M., Kamma-Lorger, C.S. (2011)  The adhesion of LASIK-like flaps in the cornea: effects of cross-linking, stromal fibroblasts and cytokine treatment.  J Cataract Refract Surg 37:166-72.
  • R.R. Mohan, J.C.K. Tovey, A. Sharma, G.S. Schultz, J.W. Cowden, A. Tandon.  Targeted decorin gene therapy delivered with adeno-associated virus effectively retards corneal neovascularization in vivo.  PLoS ONE, in press.
  • Jijing Pang, Xufeng Dai, Shannon E. Boye, Ilaria Barone, Sanford L. Boye, Song Mao, Drew Everhart, Astra Dinculescu, Li Liu, Yumiko Umino, Bo Lei, Bo Chang, Robert Barlow, Enrica Strettoi, William Hauswirth. Long term retinal function and structure rescue using capsid mutant AAV8 vector in the rd10 mouse, a model of recessive retinitis pigmentosa. Mol Ther.19:234-42. PMID: 21139570 http://www.ncbi.nlm.nih.gov/pubmed/21139570
  • Peden MC, Min J, Meyers C, Lukowski Z, Li Q, Boye SL, Levine M, Hauswirth WW, Ratnakaram R, Dawson W, Smith WC, Sherwood MB. “Ab-Externo AAV-Mediated Gene Delivery to the Suprachoroidal Space Using a 250 Micron Flexible Microcatheter.” PLoS One. 2011 Feb 11;6(2):e17140. PMID:21347253
  • Petrs-Silva H, Dinculescu A, Li Q, Deng WT, Pang JJ, Min SH, Chiodo V, Neeley AW, Govindasamy L, Bennett A, Agbandje-McKenna M, Zhong L, Li B, Jayandharan GR, Srivastava A, Lewin AS, Hauswirth WW. Novel Properties of Tyrosine-mutant AAV2 Vectors in the Mouse Retina. Molec. Ther. 19:293-301 (2011)
  • Pi L, Xia H, Liu J, Shenoy AK, Hauswirth WW, Scott EW. “The Role of Connective Tissue Growth Factor in Retinal Vasculature during Development and Ischemia” Invest Ophthalmol Vis Sci. 2011 Oct 3. [Epub ahead of print] PMID: 21969300
  • P.M. Robinson, T.D. Blalock, R. Yuan, A.S. Lewin, G.S. Schultz.  Hammerhead Ribozyme Mediated Knockdown of mRNA for Fibrotic Growth Factors: Transforming Growth Factor-Beta 1 and Connective Tissue Growth Factor. In: Cytokine Protocols, M. De Lay, Humana Press, Totowa New Jersey, in press.
  • Ruan Q, Han S, Jiang W, Boulton ME, Chen Z, Law B, Cai J.  (2011)  αB-crystallin, a effecter of unfolded protein response, confers anti-VEGF resistance to breast cancer via maintenance of intracrine VEGF in endothelial cells.  Mol Cancer Res Oct 7. [Epub ahead of print]
  • Renee C. Ryals, Sanford L. Boye, Astra Dinculescu, William W. Hauswirth, Shannon E. Boye. (2011) Quantifying transduction efficiencies of unmodified and tyrosine capsid mutant AAV vectors in vitro using two ocular cell lines. Mol Vis. 17:1090-102. PMID: 21552473 http://www.ncbi.nlm.nih.gov/pubmed/21552473
  • Simons DL, Boye SL, Hauswirth WW, Wu SM. “Gene therapy prevents photoreceptor death and preserves retinal function in a Bardet-Biedl syndrome mouse model.” Proc Natl Acad Sci U S A. 2011 Mar 28. [Epub ahead of print] PMID:2144480
  • Smith WC, Bolch SN, Dugger DR, Li J, Esquenazi I, Arendt A, Benzenhafer D, McDowell JH (2011) Interaction of arrestin with enolase1 in photoreceptors.  Invest. Ophthalmol. Vis. Sci. 52(3):1832-40
  • Soustek, MS, Falk, D, Mah, C, Toth, M, Schlame, M, Lewin, A, Byrne, B. Characterization of a Transgenic shRNA Induced Murine Model of Tafazzin Deficiency, Hum. Gene Ther. 22:567-575 (2011)
  • Steindl-Kuscher K, Boulton ME, Haas P, Dossenbach-Glaninger A, Feichtinger H, Binder S.   (2011)  Epidermal growth factor: the driving force in initiation of RPE cell proliferation.  Graefes Arch Clin Exp Ophthalmol. 249(8):1195-200.
  • Taylor S, Calder CJ, Albon J, Erichsen JT, Boulton ME, Morgan JE. (2011)  Involvement of the CD200 receptor complex in microglia activation in experimental glaucoma.  Exp Eye Res. 92(5):338-43.
  • Tuo J, Pang JJ, Cao X, Shen D, Zhang J, Scaria A, Wadsworth SC, Pechan P, Boye SL, Hauswirth WW, Chan CC. AAV5-mediated sFLT01 gene therapy arrests retinal lesions in Ccl2(-/-)/Cx3cr1(-/-) mice. Neurobiol Aging. 2011 Mar 11 [Epub ahead of print doi:10.1016/j.neurobiolaging.2011.01.009].
  • Verma A, Shan Z, Lei B, Yuan L, Liu X, Nakagawa T, Grant MB, Lewin AS, Hauswirth WW, Raizada MK, Li Q.  ACE2 and Ang-(1-7) Confer Protection Against Development of Diabetic Retinopathy Mol Ther. 2011 Jul 26. doi: 10.1038/mt.2011.155. [Epub ahead of print]
  • Verrier JD, Madorsky I, Coggin WE, Geesey M, Hochman M, et al. (2011) Bicistronic Lentiviruses Containing a Viral 2A Cleavage Sequence Reliably Co-Express Two Proteins and Restore Vision to an Animal Model of LCA1. PLoS ONE 6(5): e20553. doi:10.1371/journal.pone.0020553 http://www.plosone.org/article/info:doi/10.1371/journal.pone.0020553
  • Wang H, Murphy R, Taaffe D, Yin S, Xia L, Hauswirth WW, Bance M, Robertson GS, Wang J. “Efficient cochlear gene transfection in guinea-pigs with adeno-associated viral vectors by partial digestion of round window membrane.”Gene Ther. 2011 Jun 23. [Epub ahead of print] PMID:21697953
  • Xia Li, Wensheng Li, Xufeng Dai, Fansheng Kong, Qinxiang Zheng, Bo Chang, Barbell Rohrer, William W. Hauswirth, Jia Qu, Ji-jing Pang. AAV-mediated Cone Rescue in the Three-Month-Old rd12 Mouse: A Model for Midcourse RPE65 Leber Congenital Amaurosis. IOVS 2011; 52(1):7-15..
  •  Zou J, Luo L, Shen Z, Chiodo VA, Ambati BK, Hauswirth WW, Yang J. “Whirlin Replacement Restores the Formation of the USH2 Protein Complex in Whirlin Knockout Photoreceptors.” Invest Ophthalmol Vis Sci. 2011 Jan 6. [Epub ahead of print] PMID: 21212183

2010 Center Research

  •  Banin E, Bandah-Rosenfeld D, Obolensky A, Cideciyan AV, Aleman TS, Marks-Ohana D, Sela M, Boye SL, Sumaroka A, Roman AJ, Schwartz SB, Hauswirth W, Jacobson SG, Hemo I, Sharon D. “Molecular Anthropology meets Genetic Medicine to Treat Blindness in the North African Jewish Population: Human Gene Therapy Initiated in Israel” Hum Gene Ther. 21:1749-1757 2010 PMID: 20604683
  • Beattie JR, Pawlak AM, Boulton ME, Zhang J, Monnier VM, McGarvey JJ, Stitt AW.  (2010)  Multiplex analysis of age-related protein and lipid modifications in human Bruch’s membrane. FASEB J. 24:4816-24.
  • Beltran WA, Boye SL, Boye SE, Chiodo VA, Acland GM, Lewin AS, Hauswirth WW, Aguirre GD, rAAV2/5 Gene-Targeting to Rods: Dose-Dependent Efficiency and Side-Effects Associated With Different Promoters, Gene Therapy, 17:1162-74 (2010)
  • William A. Beltran, Sanford L. Boye, Shannon E. Boye, Vince A. Chiodo, Gregory M. Acland, Alfred S. Lewin, William W. Hauswirth, Gustavo D. Aguirre. (2010) rAAV2/5 gene-targeting to rods: dose-dependent efficiency and complications associated with different promoters. Gene Therapy. 17:1162-74. http://www.ncbi.nlm.nih.gov/pubmed/20428215
  • Shannon E. Boye, Jijing Pang, Bo Lei, Sanford L. Boye, Drew Everhart, Yumiko Umino, Baerbel Rohrer, John Alexander, Jie Li, Bo Chang, Robert Barlow, and William W. Hauswirth. (2010) Self complimentary AAV-mediated gene therapy restores cone function and prevents cone degeneration in two models of RPE65 deficiency. Gene Therapy. 17:815-26. PMID: 20237510 http://www.ncbi.nlm.nih.gov/pubmed/20237510
  • Shannon E. Boye, Sanford L. Boye, Jijing Pang, Renee Ryals, Drew Everhart, Yumiko Umino, Andy W. Neeley, Joseph Besharse, Robert Barlow, William W. Hauswirth.(2010) Functional and behavioral restoration of vision by gene therapy in a mouse model of Leber congenital amaurosis-1 (LCA1). PLoS ONE. 5: e11306. PMID: 20593011 http://www.ncbi.nlm.nih.gov/pubmed/20593011
  • Caruso RC, Aleman T, Cideciyan AV, Roman AJ, Sumaroka A, Mullins CL, Boye SL, Hauswirth WW, Jacobson SG. “Retinal Disease in Rpe65-deficient Mice: Comparison to Human Leber Congenital Amaurosis due to RPE65 mutations.”Invest Ophthalmol Vis Sci. 51:5304-5313 2010 PMID: 20484585
  • Fansheng Kong, Wensheng Li, Xia Li, Qinxiang Zheng,  Xufeng Dai,  Xiangtian Zhou, Sanford L BoyeWilliam W Hauswirth,  Jia Qu, Ji-jing Pang. Self-complementary AAV5 Vector Facilitates Quicker Transgene Expression in Photoreceptor and Retinal Pigment Epithelial Cells of Normal Mouse. Exp Eye Res. 2010; 90 (5): 546-554.
  • Gorbatyuk, MS, Knox, T, LaVail, MM, Gorbatyuk, OS, Noorwez,S M, Hauswirth, WW, Lin, JH,  Muzyczka, N. and Lewin, AS. Restoration of Visual Function in P23H Rhodopsin Transgenic Rats by Gene Delivery of BiP/Grp78. (2010) PNAS 107:5961-6. PMID: 20231467
  • Guy, J, Qi, X,  Koilkonda, RD,  Arguello, T, Chou, T-H, Porciatti, V, Lewin, AS and Hauswirth WW “.nduction of rapid and highly efficient expression of the human ND4 complex I subunit in the mouse visual system by self-complementary adeno-associated virus. ” Arch. Ophthal. 128:876-883 (2010).
  • Jarrett SG, Lewin AS, Boulton ME. (2010)  The importance of mitochondria in age-related and inherited eye disorders. Ophthalmic Res. 44(3):179-90.
  • Koilkonda RD, Chou TH, Porciatti V, Hauswirth WW, Guy J.”Induction of rapid and highly efficient expression of the human ND4 complex I subunit in the mouse visual system by self-complementary adeno-associated virus.”Arch Ophthalmol. 2010 28:876-83.PMID: 20625049
  • Komáromy AM, Alexander JJ, Rowlan JS, Garcia MM, Chiodo VA, Kaya A, Tanaka JC, Acland GM, Hauswirth WW, Aguirre GD. “Gene therapy rescues cone function in congenital achromatopsia Hum Mol Genet. 19:2581-2593 (2010) PMID: 20378608
  • Kong F, Li W, Li X, Zheng Q, Dai X, Zhou X, Boye SL, Hauswirth WW, Qu J and Pang JJ. “Self-complementary AAV5 Vector Facilitates Quicker Transgene Expression in Photoreceptor and Retinal Pigment Epithelial Cells of Normal Mouse.” Exp Eye Res. 90:546-554 (2010)  PMID 20138034
  • Lam BL, Feuer WJ, Abukhalil F, Porciatti V, Hauswirth WW, Guy J. “Leber Hereditary Optic Neuropathy Gene Therapy Clinical Trial Recruitment: Year 1.”Arch Ophthalmol. 128:1129-1135 2010 PMID: 20837795
  •  Li, Q, Han, PY, Verma, A, Nakagawa, T, Johnson, RJ, Grant, MB, Campbell-Thompson, M, Atkinson, MA, Sebal, MS and Hauswirth, WW “Diabetic eNOS knockout mice develop accelerated retinopathy” Invest Ophthalmol Vis Sci. 51:5240-5246 (2010). PMID: 20435587
  • Lobanova, Herrmann, R, Finkelstein, S, Reidel, RB, Skiba1, NP, Deng, WT, Jo, R, Weiss, ER, Hauswirth, WW and Arshavsky, VY. “Mechanistic basis for the failure of cone transducin to translocate: why cones are never blinded by light” J. Neurosci.  30:6815-24 (2010). PMID: 20484624
  •  Lukason M, Dufresne E, Rubin H, Pechan P, Li Q, Kim I, Kiss S, Flaxel C, Collins M, Miller J, Hauswirth W, Maclachlan T, Wadsworth S, Scaria A. “Inhibition of Choroidal Neovascularization in a Nonhuman Primate Model by Intravitreal Administration of an AAV2 Vector Expressing a Novel Anti-VEGF Molecule.”Mol Ther. 19:260-265 2010 PMID: 20978476
  • Orisme W, Goldmann T, Li J, Bolch S, Wolfrum U, Smith WC (2010) Arrestin translocation in rod photoreceptors is signaled through phospholipase C and is an ATP-dependent process.  Cellular Signaling 22:447-456. PMC279496
  • Ji-jing Pang, John Alexander, Bo Lei, Wentao Deng, Keqing Zhang, Qiuhong Li, Bo Chang, William W. Hauswirth. Achromatopsia as a Potential Candidate for Gene Therapy. Adv Exp Med Biol.2010; 664: 639-46 (Springer: Indexed Book Chapter).
  • Jijing Pang, Shannon E. Boye, Bo Lei, Sanford L. Boye, Drew Everhart, Yumiko Umino, Bärbel Rohrer, John Alexander, Jie Li, Xufeng Dai, Qiuhong Li, Bo Chang, Robert Barlow, and William W. Hauswirth . Early gene therapy confers structural and functional rescue to cones in two models of RPE65 deficiency: rd12 and Rpe65-/-Rho-/- mice. Gene Ther. 2010; 17, 815–826.
  • Petrs-Silva H, Dinculescu A, Li Q, Min SH, Chiodo V, Pang JJ, Zhong L, Zolotukhin S, Srivastava A, Lewin AS, Hauswirth WW. High-efficiency transduction of the mouse retina by tyrosine-mutant AAV serotype vectors. Mol Ther. 17(3):463-71. (2010)
  • Hilda Petrs-Silva, Astra Dinculescu, Qiuhong Li, Wen-Tao Deng, Ji-jing Pang, Seok-Hong Min,Vince Chiodo, Andy W. Neeley, Lakshmanan Govindasamy, Antonette Bennett,Mavis Agbandje-McKenna, Li Zhong, Baozheng Li, Giridhara R. Jayandharan,Arun Srivastava, Alfred S. Lewin and William W. Hauswirth. Novel Properties of Tyrosine-mutant AAV2 Vectors in the Mouse Retina. Mol Ther 2010; doi:10.1038/mt.2010.234
  • Expression characteristics of dual-promoter lentiviral vectors targeting retinal photoreceptors and Müller cells. Semple-Rowland SL, Coggin WE, Geesey M, Eccles KS, Abraham L, Pachigar K, Ludlow R, Khani SC, Smith WC. Mol Vis. 2010 May 27;16:916-34. http://www.molvis.org/molvis/v16/a102/index.html
  • Sengupta N, Afzal A, Caballero S, Chang KH, Shaw LC, Pang JJ, Bond VC, Bhutto I, Baba T, Lutty G, Grant MB. Paracrine Modulation of CXCR4 by IGF-1 and VEGF: Implications for Choroidal Neovascularization. Invest. Ophthalmol Vis. Sci. 2010; 51(5):2697-704.
  • Västinsalo H, Jalkanen R, Dinculescu A, Isosomppi J, Geller S, Flannery JG, Hauswirth WW, Sankila EM. “Alternative splice variants of the USH3A gene Clarin 1 (CLRN1)”Eur J Hum Genet. 19:30-35 2010 PMID: 20717163
  • Yao J, Feathers K, Khanna H, Thompson D, Tsilfidis C, Hauswirth WW, Heckenlively JR, Swaroop A, Zacks DN “XIAP Therapy Increases Survival of Transplanted Rod Precursors in a Degenerating Host Retina.”Invest Ophthalmol Vis Sci. 2010 Oct 6. [Epub ahead of print] PMID: 20926819