Intra-Articular AAV9 α-Iduronidase Gene Therapy in the Canine Model of Mucopolysaccharidosis Type I Results in Rapid Synovial and Cartilage Iduronidase Expression, Clearance of Heparan Sulfate, and High Serum α-Iduronidase Levels

被引：0

作者：

Kan, Shih-hsin ^{[1
]}

Zhang, Haoyue ^{[2
]}

Smith, Jodi D. ^{[3
]}

Snella, Elizabeth M. ^{[3
]}

Aminian, Afshin ^{[4
]}

Jens, Jackie K. ^{[3
]}

Dickson, Patricia I. ^{[5
]}

Ellinwood, N. Matthew ^{[3
]}

Young, Sarah ^{[2
]}

Wang, Raymond Y. ^{[6
]}

机构：

[1] CHOC Childrens Hosp, Dept Res Adm, Orange, CA USA

[2] Duke Univ, Dept Pediat, Sch Med, Durham, NC 27706 USA

[3] Iowa State Univ, Dept Anim Sci, Ames, IA USA

[4] CHOC Childrens Hosp, Orthoped Inst, Orange, CA USA

[5] Washington Univ, Dept Pediat, Sch Med St Louis, St Louis, MO 63130 USA

[6] CHOC Childrens Specialists, Div Metab Disorders, Orange, CA USA

来源：

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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：

945

引用

页码：435 / 436

页数：2