AAV5-miHTT gene therapy for Huntington disease: lowering both huntingtins

被引：10
|
作者：
Evers, Melvin M. [1 ]
Konstantinova, Pavlina [1 ]
机构：
[1] UniQure Biopharma BV, Res, Amsterdam, Netherlands
来源：
EXPERT OPINION ON BIOLOGICAL THERAPY | 2020年 / 20卷 / 10期
关键词：
Huntington disease; huntingtin lowering; HTT; antisense oligonucleotide; ASO; adeno-associated virus; AAV5; microRNA; EMBRYONIC LETHALITY; SUPPRESSION; MODEL; INACTIVATION; IMPROVEMENT; APOPTOSIS; PROTEIN; RNA;
D O I：
10.1080/14712598.2020.1792880
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：1121 / 1124
页数：4
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