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Page 1
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, Ye GJ, Knop DR, Robinson PM, Chulay JD, Shearman MS. Gootwine E, et al. Among authors: knop dr. Hum Gene Ther Clin Dev. 2017 Jun;28(2):96-107. doi: 10.1089/humc.2017.028. Epub 2017 May 5. Hum Gene Ther Clin Dev. 2017. PMID: 28478700
Safety and Biodistribution Evaluation in Cynomolgus Macaques of rAAV2tYF-CB-hRS1, a Recombinant Adeno-Associated Virus Vector Expressing Retinoschisin.
Ye GJ, Budzynski E, Sonnentag P, Miller PE, Sharma AK, Ver Hoeve JN, Howard K, Knop DR, Neuringer M, McGill T, Stoddard J, Chulay JD. Ye GJ, et al. Among authors: knop dr. Hum Gene Ther Clin Dev. 2015 Sep;26(3):165-76. doi: 10.1089/humc.2015.076. Hum Gene Ther Clin Dev. 2015. PMID: 26390090 Free PMC article.
Safety and Biodistribution Evaluation in CNGB3-Deficient Mice of rAAV2tYF-PR1.7-hCNGB3, a Recombinant AAV Vector for Treatment of Achromatopsia.
Ye GJ, Budzynski E, Sonnentag P, Nork TM, Miller PE, McPherson L, Ver Hoeve JN, Smith LM, Arndt T, Mandapati S, Robinson PM, Calcedo R, Knop DR, Hauswirth WW, Chulay JD. Ye GJ, et al. Among authors: knop dr. Hum Gene Ther Clin Dev. 2016 Mar;27(1):27-36. doi: 10.1089/humc.2015.163. Hum Gene Ther Clin Dev. 2016. PMID: 27003752 Free PMC article.
Safety and Biodistribution Evaluation in Cynomolgus Macaques of rAAV2tYF-PR1.7-hCNGB3, a Recombinant AAV Vector for Treatment of Achromatopsia.
Ye GJ, Budzynski E, Sonnentag P, Nork TM, Miller PE, Sharma AK, Ver Hoeve JN, Smith LM, Arndt T, Calcedo R, Gaskin C, Robinson PM, Knop DR, Hauswirth WW, Chulay JD. Ye GJ, et al. Among authors: knop dr. Hum Gene Ther Clin Dev. 2016 Mar;27(1):37-48. doi: 10.1089/humc.2015.164. Hum Gene Ther Clin Dev. 2016. PMID: 27003753 Free PMC article.
Toxicology and Pharmacology of an AAV Vector Expressing Codon-Optimized RPGR in RPGR-Deficient Rd9 Mice.
Song C, Conlon TJ, Deng WT, Coleman KE, Zhu P, Plummer C, Mandapati S, Van Hoosear M, Green KB, Sonnentag P, Sharma AK, Timmers A, Robinson PM, Knop DR, Hauswirth WW, Chulay JD, Shearman MS, Ye GJ. Song C, et al. Among authors: knop dr. Hum Gene Ther Clin Dev. 2018 Dec;29(4):188-197. doi: 10.1089/humc.2018.168. Hum Gene Ther Clin Dev. 2018. PMID: 30280954 Free PMC article.
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 VL, Cideciyan AV, Ye GJ, Song C, Timmers A, Habecker PL, Pan W, Weinstein NM, Swider M, Durham AC, Ying GS, Robinson PM, Jacobson SG, Knop DR, Chulay JD, Shearman MS, Aguirre GD, Beltran WA. Dufour VL, et al. Among authors: knop dr. Hum Gene Ther. 2020 Feb;31(3-4):253-267. doi: 10.1089/hum.2019.297. Epub 2020 Feb 6. Hum Gene Ther. 2020. PMID: 31910043 Free PMC article.
21 results