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PHARMACOLOGICAL APPROACHES FOR TARGETING CYSTIC FIBROSIS NONSENSE MUTATIONS

Cystic fibrosis (CF) is a monogenic autosomal recessive disorder. The clinical manifestations of the disease are caused by ~ 2,000 mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) protein. It is unlikely that any one approach will be efficient in correcting all defects. Th...

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Bibliografiska uppgifter
I publikationen:Eur J Med Chem
Huvudupphovsmän: Sharma, Jyoti, Keeling, Kim M., Rowe, Steven M.
Materialtyp: Artigo
Språk:Inglês
Publicerad: 2020
Ämnen:
Länkar:https://ncbi.nlm.nih.gov/pmc/articles/PMC7384597/
https://ncbi.nlm.nih.gov/pubmed/32512483
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ejmech.2020.112436
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