Intravenous delivery of AAV9-GLB1 gene therapy for GM1 gangliosidosis: An interim analysis

被引：0
|
作者：
D'Souza, Precilla [1 ]
Johnston, Jean M. [1 ]
Thurm, Audrey [2 ]
Farmer, Cristan [2 ]
Gross, Amanda L. [3 ]
Acosta, Maria T. [1 ]
Tifft, Cynthia [1 ]
机构：
[1] NHGRI, NIH, Bethesda, MD USA
[2] NIMH, NIH, Bethesda, MD USA
[3] Auburn Univ, Auburn, AL USA
来源：
MOLECULAR GENETICS AND METABOLISM | 2024年 / 141卷 / 02期
关键词：
D O I：
10.1016/j.ymgme.2023.107802
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
63
引用
收藏
页数：1
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