Phase I/II Trial of Diaphragm Delivery of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase (rAAV1-CMV-GAA) Gene Vector in Patients with Pompe Disease

被引：36

作者：

Byrne, Barry J.

Smith, Barbara

Mah, Cathryn

Lawson, Lee Ann

Islam, Saleem

Corti, Manuela

Martin, Daniel

Dobija, Nicole

Irwin, Maria V.

Conlon, Thomas

Cleaver, Brian

Clement, Nathalie

Collins, Shelley W.

机构：

[1] Univ Florida, Dept Pediat, Gainesville, FL 32610 USA

[2] Univ Florida, Dept Surg, Gainesville, FL 32610 USA

来源：

HUMAN GENE THERAPY CLINICAL DEVELOPMENT | 2014年 / 25卷 / 03期

关键词：

OBSTRUCTIVE PULMONARY-DISEASE; ADENO-ASSOCIATED VIRUS; GLYCOGENOSIS TYPE-II; SITE-SPECIFIC INTEGRATION; TRANSDUCTION IN-VIVO; SKELETAL-MUSCLE; STORAGE-DISEASE; MECHANICAL VENTILATION; MALTASE DEFICIENCY; VIRAL VECTORS;

D O I：

10.1089/humc.2014.2514

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：

[No abstract available]

引用

页码：134 / 163

页数：30

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