Allele-Specific AAV-Based Silencing of Mutant Ataxin-3 Alleviates Neuropathology and Motor Deficits in Spinocerebellar Ataxia Type 3

被引：0
|
作者：
Nobre, Rui Jorge [1 ,2 ,3 ]
Saraiva, Joana [1 ]
Santana, Magda [1 ]
Fusco, Clelia [1 ]
Paixao, Susana [1 ]
Miranda, Catarina [1 ,3 ]
Petrella, Lorena [4 ]
Sereno, Jose [4 ]
Castelhano, Joao [4 ]
Castelo-Branco, Miguel [4 ]
Sena-Esteves, Miguel [5 ]
de Almeida, Luis Pereira [1 ,2 ,6 ]
机构：
[1] Ctr Neurosci & Cell Biol, Coimbra, Portugal
[2] ViraVector Viral Vectors Gene Transfer Core Facil, Coimbra, Portugal
[3] Inst Interdisciplinary Res III, Coimbra, Portugal
[4] ICNAS Inst Nucl Sci Appl Hlth, Coimbra, Portugal
[5] Univ Massachusetts, Sch Med, Dept Neurol, Gene Therapy Ctr, Worcester, MA USA
[6] Univ Coimbra, Fac Pharm, Coimbra, Portugal
来源：
MOLECULAR THERAPY | 2020年 / 28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：
932
引用
收藏
页码：404 / 405
页数：2
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