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Viral vector-mediated and cell-based therapies for treatment of cystic fibrosis
被引:48
|作者:
Flotte, Terence R.
Ng, Philip
Dylla, Doug E.
McCray, Paul B., Jr.
Wang, Guoshun
Kolls, Jay K.
Hu, Jim
机构:
[1] Univ Toronto, Hosp Sick Children, Dept Lab Med & Pathobiol, Lung Biol Res Programme, Toronto, ON M5G 1X8, Canada
[2] Univ Florida, Dept Pediat, Gainesville, FL USA
[3] Baylor Coll Med, Dept Mol & Human Genet, Houston, TX 77030 USA
[4] Univ Iowa, Program Gene Therapy, Dept Pediat, Carver Coll Med, Iowa City, IA USA
[5] Louisiana State Univ, Hlth Sci Ctr, Dept Med, New Orleans, LA USA
[6] Louisiana State Univ, Hlth Sci Ctr, Dept Genet, New Orleans, LA USA
[7] Childrens Hosp Pittsburgh, Dept Pediat, Pittsburgh, PA 15213 USA
[8] Univ Toronto, Hosp Sick Children, Lung Biol Res Programme, Toronto, ON M5G 1X8, Canada
[9] Univ Toronto, Dept Lab Med & Pathobiol, Toronto, ON M5G 1X8, Canada
基金:
加拿大健康研究院;
美国国家卫生研究院;
关键词:
TRANSMEMBRANE CONDUCTANCE REGULATOR;
ADENO-ASSOCIATED VIRUS;
AIRWAY EPITHELIAL-CELLS;
ADULT STEM-CELLS;
HUMORAL IMMUNE-RESPONSES;
TARGETING TRANSGENE EXPRESSION;
MONKEYS MACACA-MULATTA;
GENE-TRANSFER VECTORS;
MARROW-DERIVED CELLS;
LONG-TERM EXPRESSION;
D O I:
10.1038/sj.mt.6300002
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:
Gene and cell-based therapies are considered to be potentially powerful new approaches for the management of cystic fibrosis (CF) lung disease. Despite tremendous efforts that have been made, especially in studies to understand the obstacles to gene delivery, major challenges to the application of these approaches remain to be solved. This article will review the advancements made and challenges remaining in the development of viral vector - mediated and cell-based approaches to treat patients with CF.
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页码:229 / 241
页数:13
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