- Abstract:
-
Cystic fibrosis (CF) a monogenic lethal disease and, therefore, ideally suited for the development of gene therapy. The first clinical trials were carried out shortly after cloning the CF gene in 1989. Since then, 25 trials have been carried out. Proof of principle for low-level airway gene transfer was established in most, but not all, trials. It is currently unclear whether current gene transfer efficiency will lead to improvements in clinically relevant endpoints such as inflammation or in...
Expand abstract - Publisher copy:
- 10.1007/978-1-59745-237-3_14
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- Journal:
- Methods in molecular biology (Clifton, N.J.)
- Volume:
- 433
- Pages:
- 229-242
- Publication date:
- 2008
- DOI:
- EISSN:
-
1940-6029
- ISSN:
-
1064-3745
- URN:
-
uuid:68e4a6c5-37a7-478d-9185-795aed3b0f06
- Source identifiers:
-
116812
- Local pid:
- pubs:116812
- Copyright date:
- 2008
Journal article
Assessment of CFTR function after gene transfer in vitro and in vivo.
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