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Aguirre G.D., Cideciyan A.V., Dufour V.L., Ripolles Garcia A., Sudharsan R., Swider M., Nikonov R., Iwabe S., Boye S.L., Hauswirth W.W., Jacobson S.G., Beltran W.A. Gene Therapy Reforms Photoreceptor Structure and Restores Vision in NPHP5-associated Leber Congenital Amaurosis. bioRxiv, 2020 (preprint).
Hardcastle A.J., Sieving P.A., Sahel J.A., Jacobson S.G., Cideciyan A.V., Flannery J.G., Beltran W.A., Aguirre G.D. Translational Retinal Research and Therapies. Transl Vis Sci Technol., 2018; 7(5):8.
Downs L.M., Scott E.M., Cideciyan A.V., Iwabe S., Dufour V., Gardiner K.L., Genini S., Marinho L.F., Sumaroka A., Kosyk M.S., Swider M., Aguirre G.K., Jacobson S.G., Beltran W.A., Aguirre G.D. Overlap of Abnormal Photoreceptor Development and Progressive Degeneration in Leber Congenital Amaurosis Caused by NPHP5 Mutation. Hum Mol Genet., 2016; 25 (19): 4211-4226.
Beltran W.A., Cideciyan A.V., Boye S.E., Ye G-J., Iwabe S., Dufour V., Marinho L.F., Swider M., Kosyk M., Sha J., Boye S.L., Peterson J.J., Witherspoon C.D., Alexander J.J., Ying G-S., Shearman M.S., Chulay J.D., Hauswirth W.W., Gamlin P.D., Jacobson S.G., Aguirre G.D. Optimization of retinal gene therapy for X-linked retinitis pigmentosa due to RPGR mutations. Mol Ther., 2017; 25(8): 1866-1880.
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. Successful arrest of photoreceptor and vision loss expands the therapeutic window of retinal gene therapy to later stages of disease. Proc Natl Acad Sci USA., 2015; 112(43): E5844-5853.
Beltran W.A., Cideciyan A.V., Lewin A.S., Iwabe S., Khanna H., Sumaroka A., Chiodo V.A., Fajardo D.S., Román A.J., Deng W.-T., Swider M., Alemán T.S., Boye S.L., Genini S., Swaroop A., Hauswirth W.W., Jacobson S.G., Aguirre G.D. Gene therapy rescues photoreceptor blindness in dogs and paves the way for treating human X-linked retinitis pigmentosa. Proc Natl Acad Sci USA., 2012; 109(6): 2132-2137.
Aguirre G.D., Cideciyan A.V., Dufour V.L., Ripolles Garcia A., Sudharsan R., Swider M., Nikonov R., Iwabe S., Boye S.L., Hauswirth W.W., Jacobson S.G., Beltran W.A. Gene Therapy Reforms Photoreceptor Structure and Restores Vision in NPHP5-associated Leber Congenital Amaurosis. bioRxiv, 2020 (preprint).
Hardcastle A.J., Sieving P.A., Sahel J.A., Jacobson S.G., Cideciyan A.V., Flannery J.G., Beltran W.A., Aguirre G.D. Translational Retinal Research and Therapies. Transl Vis Sci Technol., 2018; 7(5):8.
Downs L.M., Scott E.M., Cideciyan A.V., Iwabe S., Dufour V., Gardiner K.L., Genini S., Marinho L.F., Sumaroka A., Kosyk M.S., Swider M., Aguirre G.K., Jacobson S.G., Beltran W.A., Aguirre G.D. Overlap of Abnormal Photoreceptor Development and Progressive Degeneration in Leber Congenital Amaurosis Caused by NPHP5 Mutation. Hum Mol Genet., 2016; 25 (19): 4211-4226.
Beltran W.A., Cideciyan A.V., Boye S.E., Ye G-J., Iwabe S., Dufour V., Marinho L.F., Swider M., Kosyk M., Sha J., Boye S.L., Peterson J.J., Witherspoon C.D., Alexander J.J., Ying G-S., Shearman M.S., Chulay J.D., Hauswirth W.W., Gamlin P.D., Jacobson S.G., Aguirre G.D. Optimization of retinal gene therapy for X-linked retinitis pigmentosa due to RPGR mutations. Mol Ther., 2017; 25(8): 1866-1880.
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. Successful arrest of photoreceptor and vision loss expands the therapeutic window of retinal gene therapy to later stages of disease. Proc Natl Acad Sci USA., 2015; 112(43): E5844-5853.
Beltran W.A., Cideciyan A.V., Lewin A.S., Iwabe S., Khanna H., Sumaroka A., Chiodo V.A., Fajardo D.S., Román A.J., Deng W.-T., Swider M., Alemán T.S., Boye S.L., Genini S., Swaroop A., Hauswirth W.W., Jacobson S.G., Aguirre G.D. Gene therapy rescues photoreceptor blindness in dogs and paves the way for treating human X-linked retinitis pigmentosa. Proc Natl Acad Sci USA., 2012; 109(6): 2132-2137.