High level expression of human iduronidase throughout the brain in a murine model of mucopolysaccharidosis type I (MPS I) after non-invasive AAV-mediated gene delivery to the CNS

被引：0

|

作者：

Kozarsky, Karen ^{[1
]}

Nan, Zhenhong ^{[2
]}

Buckvold, Megan ^{[2
]}

Karlen, Andrea ^{[2
]}

Kitto, Kelley ^{[2
]}

Podetz-Pedersen, Kelly M. ^{[2
]}

Fairbanks, Carolyn ^{[2
]}

Hanson, Leah R. ^{[3
]}

Orchard, Paul ^{[2
]}

Tolar, Jakub ^{[2
]}

Frey, William H., II ^{[3
]}

Low, Walter C. ^{[2
]}

McIvor, R. Scott ^{[2
]}

Belur, Lalitha ^{[2
]}

机构：

[1] ReGenX Biosci, Washington, DC 20006 USA

[2] Univ Minnesota, Minneapolis, MN 55455 USA

[3] Reg Hosp, St Paul, MN 55101 USA

来源：

HUMAN GENE THERAPY | 2014年 / 25卷 / 11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：

OR052

引用

收藏

页码：A42 / A42

页数：1

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