期刊
NATURE BIOTECHNOLOGY
卷 19, 期 9, 页码 838-842出版社
NATURE AMERICA INC
DOI: 10.1038/nbt0901-838
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资金
- NCI NIH HHS [R01 CA74242] Funding Source: Medline
- NIDDK NIH HHS [P30 DK54781] Funding Source: Medline
The promise of gene therapy for health care will not be realized until gene delivery systems are capable of achieving efficient, cell-specific gene delivery in vivo. Here we describe an adenoviral system for achieving cell-specific transgene expression in pulmonary endothelium. The combination of transductional targeting to a pulmonary endothelial marker (angiotensin-converting enzyme, ACE) and an endothelial-specific promoter (for vascular endothelial growth factor receptor type 1, fit-1) resulted in a synergistic, 300,000-fold improvement in the selectivity of transgene expression for lung versus the usual site of vector sequestration, the liver. This combined approach should be useful for the design of other gene delivery systems.
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