Beltran, William A.

Gene therapy rescues photoreceptor blindness in dogs and paves the way for treating human X-linked retinitis pigmentosa

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., et al. (2012) · Proceedings of the National Academy of Sciences

Retinal Development and DisordersVirus-based gene therapy research

Cited by 260

Mutation-independent rhodopsin gene therapy by knockdown and replacement with a single AAV vector

Cideciyan, A. V., Sudharsan, R., Dufour, V. L., Massengill, M. T., Iwabe, S., Swider, M., Lisi, B., Sumaroka, A., Marinho, L. F., Appelbaum, T., et al. (2018) · Proceedings of the National Academy of Sciences

Retinal Development and DisordersVirus-based gene therapy research

Cited by 147

rAAV2/5 gene-targeting to rods:dose-dependent efficiency and complications associated with different promoters

Beltran, W. A., Boye, S. L., Boye, S. E., Chiodo, V. A., Lewin, A. S., Hauswirth, W. W., & Aguirre, G. D. (2010) · Gene Therapy

Retinal Development and DisordersVirus-based gene therapy research

Cited by 96

Optimization of Retinal Gene Therapy for X-Linked Retinitis Pigmentosa Due to RPGR Mutations

Beltran, W. A., Cideciyan, A. V., Boye, S. E., Ye, G.-J., Iwabe, S., Dufour, V. L., Marinho, L. F., Swider, M., Kosyk, M. S., Sha, J., et al. (2017) · Molecular Therapy

Retinal Development and DisordersVirus-based gene therapy research

Cited by 82

Long-term Structural Outcomes of Late-stage RPE65 Gene Therapy

Gardiner, K. L., Cideciyan, A. V., Swider, M., Dufour, V. L., Sumaroka, A., Komáromy, A. M., Hauswirth, W. W., Iwabe, S., Jacobson, S. G., Beltran, W. A., et al. (2020) · Molecular Therapy

Retinal Development and DisordersVirus-based gene therapy research

Cited by 67

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

Cited by 39

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

Cited by 28