Journal article icon

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...

Expand abstract

Actions


Access Document


Authors


Journal:
Methods in molecular biology (Clifton, N.J.) More from this journal
Volume:
433
Pages:
229-242
Publication date:
2008-01-01
DOI:
EISSN:
1940-6029
ISSN:
1064-3745
Language:
English
Keywords:
Pubs id:
pubs:116812
UUID:
uuid:68e4a6c5-37a7-478d-9185-795aed3b0f06
Local pid:
pubs:116812
Source identifiers:
116812
Deposit date:
2012-12-19

Terms of use


Views and Downloads






If you are the owner of this record, you can report an update to it here: Report update to this record

TO TOP