作者
John A Wagner, Thomas Reynolds, Mary Lynn Moran, Richard B Moss, Jeffrey J Wine, Terence R Flotte, Phyllis Gardner
发表日期
1998/6/6
期刊
The Lancet
卷号
351
期号
9117
页码范围
1702-1703
出版商
Elsevier
简介
Cystic fibrosis is a common, lethal, genetic disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR), and it is an attractive target for gene therapy. Adeno-associated virus (AAV) is a naturally replicationdeficient single-stranded DNA parvovirus. Preclinical studies showed that CFTR transcripts and protein can be detected up to 6 months after transduction with an AAV-CFTR vector, 1 with no pathogenicity, 2 suggesting that AAV performs well as a gene transfer vehicle for CFTR. The maxillary sinuses are attractive for evaluating new treatments of cystic fibrosis because they have ion-transport systems and microbiology similar to those of the lower respiratory tract. 3, 4 Recurrence of maxillary sinusitis may prove to be a surrogate for infectious exacerbations characteristic of cystic fibrosis lung disease. Ten patients who had undergone bilateral endoscopic antrostomies were …
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