AAV5-GLA Gene Therapy Results in Sustained Long Term GLA Transgene Expression and Cross Correction of Target Organs in Fabry Disease Mouse Model

被引：0
|
作者：
Liefhebber, Jolanda M. P. [1 ]
Brasser, Giso [1 ]
Revers, Ilma [1 ]
Paerels, Lieke [1 ]
Ferraz, Maria J. [2 ]
Ottenhoff, Roelof [3 ]
Schwarz, Lukas K. [1 ]
Squeri, Giorgia [1 ]
Pereira, Ines L. [1 ]
Allart, Leonie [1 ]
Tripathi, Shrijana [1 ]
Efthymiopoulou, Nikoleta [1 ]
Dobrynin, Greg [1 ]
Montenegro-Miranda, Paula S. [1 ]
de Vries, Carlie J. M. [1 ]
Aerts, Johannes M. F. G. [1 ]
Liu, Ying Poi [1 ]
机构：
[1] UniQure, Res, Amsterdam, Netherlands
[2] Leiden Univ, Leiden Inst Chem, Med Biochem, Amsterdam, Netherlands
[3] Univ Amsterdam, Med Biochem, Amsterdam UMC, Amsterdam, Netherlands
来源：
MOLECULAR THERAPY | 2022年 / 30卷 / 04期
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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：
1009
引用
收藏
页码：472 / 472
页数：1
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