Knop, David R.

Phase 2 Clinical Trial of a Recombinant Adeno-Associated Viral Vector Expressing α 1 -Antitrypsin: Interim Results

Flotte, T. R., Trapnell, B. C., Humphries, M., Carey, B., Calcedo, R., Rouhani, F., Campbell-Thompson, M., Yachnis, A. T., Sandhaus, R. A., McElvaney, N. G., et al. (2011) · Human Gene Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

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Human Treg responses allow sustained recombinant adeno-associated virus–mediated transgene expression

Mueller, C., Chulay, J. D., Trapnell, B. C., Humphries, M., Carey, B., Sandhaus, R. A., McElvaney, N. G., Messina, L., Tang, Q., Rouhani, F. N., et al. (2013) · Journal of Clinical Investigation

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 169

Ocular Inflammatory Response to Intravitreal Injection of Adeno-Associated Virus Vector: Relative Contribution of Genome and Capsid

Timmers, A. M., Newmark, J. A., Turunen, H. T., Farivar, T., Liu, J., Song, C., Ye, G.-J., Pennock, S., Gaskin, C., Knop, D. R., et al. (2020) · Human Gene Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 77

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

Cited by 64

Safety and Biodistribution Evaluation in Cynomolgus Macaques of rAAV2tYF-CB-hRS1, a Recombinant Adeno-Associated Virus Vector Expressing Retinoschisin

Ye, G.-J., Budzynski, E., Sonnentag, P., Miller, P. E., Sharma, A. K., Ver Hoeve, J. N., Howard, K., Knop, D. R., Neuringer, M., McGill, T., et al. (2015) · Human Gene Therapy Clinical Development

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 53

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

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

Cited by 36

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

Herpes Simplex Virus Clearance During Purification of a Recombinant Adeno-Associated Virus Serotype 1 Vector

Ye, G.-J., Scotti, M. M., Thomas, D. L., Wang, L., Knop, D. R., & Chulay, J. D. (2014) · Human Gene Therapy Clinical Development

Virus-based gene therapy researchProtein purification and stability

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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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