Fixing cystic fibrosis by correcting CFTR domain assembly.
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ABSTRACT:
SUBMITTER: Okiyoneda T
PROVIDER: S-EPMC3471238 | biostudies-literature | 2012 Oct
REPOSITORIES: biostudies-literature
Publications
Fixing cystic fibrosis by correcting CFTR domain assembly.
Okiyoneda Tsukasa T Lukacs Gergely L GL
The Journal of cell biology 20121001 2
For cystic fibrosis (CF) patients most therapies focus on alleviating the disease symptoms. Yet the cellular basis of the disease has been well studied; mutations in the CF gene can impair folding, secretion, cell surface stability, and/or function of the CFTR chloride channel. Correction of these basic defects has been a challenge, but indicates that a deeper understanding of the molecular and cellular mechanism of mutations is a prerequisite for developing more efficient therapies. ...[more]
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