期刊
EXPERT OPINION ON BIOLOGICAL THERAPY
卷 20, 期 10, 页码 1121-1124出版社
TAYLOR & FRANCIS LTD
DOI: 10.1080/14712598.2020.1792880
关键词
Huntington disease; huntingtin lowering; HTT; antisense oligonucleotide; ASO; adeno-associated virus; AAV5; microRNA
资金
- uniQure biopharma B.V.
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