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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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書目詳細資料
發表在:Eur J Med Chem
Main Authors: Sharma, Jyoti, Keeling, Kim M., Rowe, Steven M.
格式: Artigo
語言:Inglês
出版: 2020
主題:
在線閱讀: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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