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
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
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页码:735 / 735
页数:1
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