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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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Authors


Griesenbach, U More by this author
Munkonge, FM More by this author
Sumner-Jones, S More by this author
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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

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