Development and validation of a novel adeno-associated viral gene therapy for mucopolysaccharidosis type IIIB (MPS IIIB)

被引：0
|
作者：
Ballout, Nissrine [1 ]
Verdu, Lena [1 ]
Santiago, Christophe [1 ]
Rouahi, Myriam [1 ]
Karim, Zoubida [1 ]
Piguet, Francoise [2 ]
Ausseil, Jerome [3 ,4 ]
机构：
[1] Univ Toulouse III Paul Sabatier, CNRS UMR5051, Toulouse Inst Infect & Inflammatory Dis, INSERM UMR1291, Toulouse, France
[2] Sorbonne Univ, Paris Brain Inst ICM, Univ Hosp Pitie Salpetriere, AP HP,CNRS,TIDU GENOV, Paris, France
[3] Univ Toulouse III, CNRS UMR5051, Toulouse Inst Infect & Inflammatory Dis, INSERM UMR1291, Toulouse, France
[4] CHU Toulouse, Lab Biochem & Mol Biol, Toulouse, France
来源：
MOLECULAR GENETICS AND METABOLISM | 2023年 / 138卷 / 02期
关键词：
D O I：
10.1016/j.ymgme.2022.107019
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
36
引用
收藏
页码：13 / 13
页数：1
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