Sustained Mutant Huntingtin Lowering in the Brain and Cerebrospinal Fluid of Huntington Disease Minipigs Mediated by AAV5-miHTT Gene Therapy

被引：0

作者：

Valles, Astrid ^{[1
]}

Stam, Anouk ^{[1
]}

Brouwers, Cynthia ^{[1
]}

Klima, Jiri ^{[2
]}

Bohuslavova, Bozena ^{[2
]}

Pintauro, Roberta ^{[3
]}

Sogorb-Gonzalez, Marina ^{[1
]}

Paerels, Lieke ^{[1
]}

Fodale, Valentina ^{[3
]}

Bresciani, Alberto ^{[3
]}

Ellederova, Zdenka ^{[2
]}

Blits, Bas ^{[1
]}

Motlik, Jan ^{[2
]}

van Deventer, Sander ^{[1
]}

Evers, Melvin ^{[1
]}

Konstantinova, Pavlina ^{[1
]}

机构：

[1] UniQure, Dept Res & Dev, Amsterdam, Netherlands

[2] Inst Anim Physiol & Genet, Libechov, Czech Republic

[3] IRBM Sci Pk, Pomezia, Italy

来源：

MOLECULAR THERAPY | 2019年 / 27卷 / 04期

关键词：

D O I：

暂无

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：

672

引用

页码：316 / 316

页数：1

共 50 条

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