RGX-121 Gene Therapy for the Treatment of Neuronopathic Mucopolysaccharidosis Type II (MPS II): Interim Analysis of Data from the First in Human Study

被引：0
|
作者：
Harmatz, Paul [1 ]
Ficicioglu, Can [2 ]
Giugliani, Roberto [3 ]
Rajan, Deepa [4 ]
Hagood, Joseph [5 ]
Fiscella, Michele [5 ]
Yang, Lin [6 ]
Gilmor, Michelle [5 ]
Cho, Yoonjin [5 ]
Phillips, Dawn [5 ]
Pisani, Laura [5 ]
Falabella, Paulo [5 ]
机构：
[1] UCSF, Benioff Childrens Hosp Oakland, Oakland, CA USA
[2] Childrens Hosp Philadelhia, Philadelphia, PA USA
[3] Hosp Clin Porto Alegre, Porto Alegre, Brazil
[4] Univ Pittsburgh, Pittsburgh, PA USA
[5] REGENXBIO Inc, Rockville, MD USA
[6] REGENXBIO, Rockville, MD USA
来源：
MOLECULAR THERAPY | 2023年 / 31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：
807
引用
收藏
页码：398 / 398
页数：1
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