Robinson, Paulette M.

Safety and biodistribution evaluation in cynomolgus macaques of rAAV2tYF-PR1. 7-hCNGB3, a recombinant AAV vector for treatment of achromatopsia

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., et al. (2016) · Human Gene Therapy Clinical Development

Retinal Development and DisordersVirus-based gene therapy research

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Toxicity and Efficacy Evaluation of an Adeno-Associated Virus Vector Expressing Codon-Optimized RPGR Delivered by Subretinal Injection in a Canine Model of X-linked Retinitis Pigmentosa

Dufour, V. L., Cideciyan, A. V., Ye, G.-J., Song, C., Timmers, A., Habecker, P. L., Pan, W., Weinstein, N. M., Swider, M., Durham, A. C., et al. (2020) · Human Gene Therapy

Retinal Development and DisordersVirus-based gene therapy research

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Safety and Efficacy Evaluation of rAAV2tYF-PR1.7-hCNGA3 Vector Delivered by Subretinal Injection in CNGA3 Mutant Achromatopsia Sheep

Gootwine, E., Ofri, R., Banin, E., Obolensky, A., Averbukh, E., Ezra-Elia, R., Ross, M., Honig, H., Rosov, A., Yamin, E., et al. (2017) · Human Gene Therapy Clinical Development

Retinal Development and DisordersVirus-based gene therapy research

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Dose Range Finding Studies with Two RPGR Transgenes in a Canine Model of X-Linked Retinitis Pigmentosa Treated with Subretinal Gene Therapy

Song, C., Dufour, V. L., Cideciyan, A. V., Ye, G.-J., Swider, M., Newmark, J. A., Timmers, A. M., Robinson, P. M., Knop, D. R., Chulay, J. D., et al. (2020) · Human Gene Therapy

Retinal Development and DisordersVirus-based gene therapy research

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Toxicology and pharmacology of an AAV vector expressing codon-optimized RPGR in RPGR-deficient Rd9 mice

Song, C., Conlon, T. J., Deng, W.-T., Coleman, K. E., Zhu, P., Plummer, C., Mandapati, S., Van Hoosear, M., Green, K. B., Sonnentag, P., et al. (2018) · Human Gene Therapy Clinical Development

Retinal Development and DisordersVirus-based gene therapy research

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