Journal article
Assessment of CFTR function after gene transfer in vitro and in vivo.
- 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...
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Bibliographic Details
- Journal:
- Methods in molecular biology (Clifton, N.J.)
- Volume:
- 433
- Pages:
- 229-242
- Publication date:
- 2008-01-01
- DOI:
- EISSN:
-
1940-6029
- ISSN:
-
1064-3745
- Source identifiers:
-
116812
Item Description
- Language:
- English
- Keywords:
- Pubs id:
-
pubs:116812
- UUID:
-
uuid:68e4a6c5-37a7-478d-9185-795aed3b0f06
- Local pid:
- pubs:116812
- Deposit date:
- 2012-12-19
Terms of use
- Copyright date:
- 2008
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