Safety and Biodistribution Evaluation in Cynomolgus Macaques of rAAV2tYF-PR1.7-hCNGB3, a Recombinant AAV Vector for Treatment of Achromatopsia.

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作者： Guo-jie,Ye ^[1] ; Ewa,Budzynski ^[2] ; Peter,Sonnentag ^[2] ; T Michael,Nork ^[3] ; Paul E,Miller ^[3] ; Alok K,Sharma ^[2] ; James N,Ver Hoeve ^[3] ; Leia M,Smith ^[4] ; Tara,Arndt ^[2] ; Roberto,Calcedo ^[5] ; Chantelle,Gaskin ^[1] ; Paulette M,Robinson ^[1] ; David R,Knop ^[1] ; William W,Hauswirth ^[6] ; Jeffrey D,Chulay ^[1]

作者单位： 1 Applied Genetic Technologies Corporation (AGTC) , Alachua, FL. ^[1] 2 Covance Laboratories , Madison, WI. ^[2] 3 OSOD (Ocular Services On Demand) , Madison, WI. ^[3] 4 Laboratory Corporation of America Holdings , Seattle, WA. ^[4] 5 University of Pennsylvania , Philadelphia, PA. ^[5] 6 University of Florida , Gainesville, FL. ^[6]

主题词动物(Animals);色觉缺陷(Color Vision Defects);环核苷酸门控阳离子通道(Cyclic Nucleotide-Gated Cation Channels);DNA, 重组(DNA, Recombinant);依赖病毒(Dependovirus);女(雌)性(Female);遗传载体(Genetic Vectors);人类(Humans);注射, 眼内(Injections, Intraocular);成束猴(Macaca fascicularis);男(雄)性(Male)

DOI 10.1089/humc.2015.164

PMID 27003753

发布时间 2018-11-13

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Human gene therapy. Clinical development

2016年27卷1期

37-48页

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Safety and Biodistribution Evaluation in Cynomolgus Macaques of rAAV2tYF-PR1.7-hCNGB3, a Recombinant AAV Vector for Treatment of Achromatopsia.

Human gene therapy. Clinical development

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Safety and Biodistribution Evaluation in Cynomolgus Macaques of rAAV2tYF-PR1.7-hCNGB3, a Recombinant AAV Vector for Treatment of Achromatopsia.

Human gene therapy. Clinical development

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Human gene therapy. Clinical development

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