Birth of a new therapeutic platform: 47 years of adeno-associated virus biology from virus discovery to licensed gene therapy.
作者信息
Flotte Terence R
机构信息
Gene Therapy Center, Department of Pediatrics, University of Massachusetts Medical School, Worcester, Massachusetts, USA.
出版信息
Mol Ther. 2013 Nov;21(11):1976-81. doi: 10.1038/mt.2013.226.
Abstract
摘要
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Three-year follow-up after unilateral subretinal delivery of adeno-associated virus in patients with Leber congenital Amaurosis type 2.2 型莱伯先天性黑矇患者单侧视网膜下注射腺相关病毒后的 3 年随访结果。
Ophthalmology. 2013 Jun;120(6):1283-91. doi: 10.1016/j.ophtha.2012.11.048. Epub 2013 Mar 6.
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