Birth of a New Therapeutic Platform: 47 Years of Adeno-associated Virus Biology From Virus Discovery to Licensed Gene Therapy

被引:16
作者
Flotte, Terence R. [1 ]
机构
[1] Univ Massachusetts, Sch Med, Dept Pediat, Gene Therapy Ctr, Worcester, MA USA
关键词
ADENO-ASSOCIATED VIRUS; LEBER CONGENITAL AMAUROSIS; PHASE-I TRIAL; TRANSMEMBRANE CONDUCTANCE REGULATOR; CYSTIC-FIBROSIS; MESSENGER-RNA; CLINICAL-TRIAL; WILD-TYPE; INTRAMUSCULAR INJECTION; RECOMBINANT PLASMID;
D O I
10.1038/mt.2013.226
中图分类号
Q81 [生物工程学(生物技术)]; Q93 [微生物学];
学科分类号
071005 ; 0836 ; 090102 ; 100705 ;
摘要
引用
收藏
页码:1976 / 1981
页数:6
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