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

页码：398 / 398

页数：1

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