AAV-Mediated PPT1 Replacement and Cross Correction for Treating CLN1 Batten Disease

被引：0
|
作者：
Alam, Suhail [1 ]
Khatiwada, Apeksha [1 ]
Cohen, Daniel M. [1 ]
Peterson, Drew R. [1 ]
White, John [1 ]
Derby, Melissa [1 ]
Wang, Bo [1 ]
Nayal, Mohamad [1 ]
Becker, Mallory [1 ]
Benakanakere, Manju [1 ]
Beck, Heena [1 ]
Li, Charlie [1 ]
Gentzel, Renee [1 ]
McBride, Jodi L. [1 ]
Biferi, Maria Grazia [1 ]
Ramsburg, Elizabeth [1 ]
机构：
[1] Spark Therapeut, Philadelphia, PA USA
来源：
MOLECULAR THERAPY | 2024年 / 32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：
1575
引用
收藏
页码：735 / 735
页数：1
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