期刊
GENE THERAPY
卷 16, 期 9, 页码 1059-1065出版社
NATURE PUBLISHING GROUP
DOI: 10.1038/gt.2009.87
关键词
gene transfer; lung; re-administration
类别
资金
- NATIONAL INSTITUTE OF DIABETES AND DIGESTIVE AND KIDNEY DISEASES [P30DK054759] Funding Source: NIH RePORTER
- NIDDK NIH HHS [P30 DK054759] Funding Source: Medline
Pulmonary gene therapy may ultimately cure diseases such as cystic fibrosis, alpha 1-antitrypsin deficiency, lung cancer and pulmonary hypertension. Efficient expression of delivered genes in target cell types is essential for the achievement of this goal. To this end, re-administration of viral vectors may be required (1) to increase the percentage of transduced airway epithelial cells, (2) to direct gene transfer to individual lobes during successive delivery sessions or (3) to boost attenuated expression over time. Immune responses to viral proteins or viral-encoded proteins are the greatest barrier to repeated vector administration. Gene Therapy (2009) 16, 1059-1065; doi: 10.1038/gt.2009.87; published online 30 July 2009
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