Efficacy of AAV5-GLA gene therapy in manifest Fabry disease mice

被引：0

作者：

Liefhebber, Jolanda M. P. ^{[1
]}

Brasser, Giso ^{[1
]}

Ferraz, Maria J. ^{[2
]}

Ottenhoff, Roelof ^{[3
]}

Efthymiopoulou, Nikoleta ^{[1
]}

Paerels, Lieke ^{[1
]}

Pereira, Ines ^{[1
]}

Sonea, Andra ^{[1
]}

Allart, Leonie ^{[1
]}

Schwarz, Lukas K. ^{[1
]}

Squeri, Giorgia ^{[1
]}

Dobrynin, Greg ^{[1
]}

Rodriguez, Rosa Crespo ^{[1
]}

Liu, Ying Poi ^{[1
]}

Aerts, Johannes M. F. G. ^{[2
]}

Montenegro-Miranda, Paula S. ^{[1
]}

机构：

[1] UniQure Biopharma BV, Amsterdam, Netherlands

[2] Leiden Univ, Leiden, Netherlands

[3] Univ Amsterdam, Amsterdam UMC, Amsterdam, Netherlands

来源：

MOLECULAR GENETICS AND METABOLISM | 2023年 / 138卷 / 02期

关键词：

D O I：

10.1016/j.ymgme.2022.107206

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

223

引用

页码：81 / 82

页数：2

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