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Journal article

Self-reactive CFTR T cells in humans: implications for gene therapy.

Abstract:

Cystic fibrosis (CF) is one of the most common autosomal recessive lethal disorders affecting white populations of northern European ancestry. To date there is no cure for CF. Life-long treatments for CF are being developed and include gene therapy and the use of small-molecule drugs designed to target specific cystic fibrosis transmembrane conductance regulator (CFTR) gene mutations. Irrespective of the type of molecular therapy for CF, which may include gene replacement, exon skipping, nons...

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Publication status:
Published

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Publisher copy:
10.1089/humc.2012.249

Authors


Journal:
Human gene therapy. Clinical development More from this journal
Volume:
24
Issue:
3
Pages:
108-115
Publication date:
2013-09-01
DOI:
EISSN:
2324-8645
ISSN:
2324-8637
Language:
English
Keywords:
Pubs id:
pubs:462860
UUID:
uuid:8b087199-bb7a-4189-958f-37ce755a20e0
Local pid:
pubs:462860
Source identifiers:
462860
Deposit date:
2014-08-22

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