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JAMA Clinical Reviews

Matching Drugs to Genetic Abnormalities to Precisely Treat Cystic Fibrosis

26 min5 december 2017

Cystic fibrosis is a common autosomal recessive disease. It is caused by any one of many discrete genetic abnormalities that affect chloride transport. Identification of specific genetic abnormalities enables clinicians to identify drugs that counteract the effects of the abnormal genes. In this podcast we review how genetic defects that cause cystic fibrosis are identified and how drugs that are likely to successfully treat the disease are matched to those genetic abnormalities.

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