Article
Cystic fibrosis transmembrane regulator protein mutations: 'class' opportunity for novel drug innovation.
Paediatric drugs - 1 Jan 2007
MacDonald Kelvin D, McKenzie Karen R, Zeitlin Pamela L
Abstract excerpt
Cystic fibrosis (CF) is the most common autosomal, recessive, life-span shortening disease in Caucasians. Since discovery of the gene for CF (cystic fibrosis transmembrane conductance regulator [CFTR]) in 1989, knowledge of the molecular function of this gene and its interactions has offered new therapeutic targets. New therapeutics aimed at improving mutant CFTR protein function, also known as 'protein repair...
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