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

被引：0
|
作者：
GHiugliani, Roberto [1 ]
Escolar, Maria [2 ]
Ficicioglu, Can [3 ]
Harmatz, Paul [4 ]
Fiscella, Michele [5 ]
Yang, Lin [5 ]
Cho, Yoonjin [5 ]
Phillips, Dawn [5 ]
Falabella, Paulo [5 ]
机构：
[1] Univ Fed Rio Grande do Sul, HCPA, Med Genet Serv, Dept Genet, Porto Alegre, RS, Brazil
[2] Univ Pittsburgh, Pittsburgh, PA USA
[3] Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA
[4] UCSF Benioff Childrens Hosp, Oakland, CA USA
[5] REGENXBIO, Rockville, MD USA
来源：
MOLECULAR THERAPY | 2022年 / 30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：
52
引用
收藏
页码：27 / 27
页数：1
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