Genetics of cystic fibrosis: CFTR mutation classifications toward genotype-based CF therapies.Cystic fibrosis (CF) is an autosomal recessive disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which encodes an epithelial anion channel.  The identification of the CFTR gene in 1989 gave hopes of rapidly finding a cure for the disease, for which over 1950 mutations have been identified.  Since 2012, recent approaches have enabled the identification of small molecules targeting either the CFTR protein directly or its key processing steps, giving rise to novel promising therapeutic tools.  This review presents the current CFTR mutation classifications according to their clinical consequences and to their effect on the structure and function of the CFTR channel.  The future of CFTR-targeted treatment lies in combinatory therapies that will enable CF patients to receive a customized treatment.
CFTR
Genetics of cystic fibrosis: CFTR mutation classifications toward genotype-based CF therapies.Cystic fibrosis (CF) is an autosomal recessive disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which encodes an epithelial anion channel.  The identification of the CFTR gene in 1989 gave hopes of rapidly finding a cure for the disease, for which over 1950 mutations have been identified.  Since 2012, recent approaches have enabled the identification of small molecules targeting either the CFTR protein directly or its key processing steps, giving rise to novel promising therapeutic tools.  This review presents the current CFTR mutation classifications according to their clinical consequences and to their effect on the structure and function of the CFTR channel.  The future of CFTR-targeted treatment lies in combinatory therapies that will enable CF patients to receive a customized treatment.
CFTR
Genetics of cystic fibrosis: CFTR mutation classifications toward genotype-based CF therapies.Cystic fibrosis (CF) is an autosomal recessive disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which encodes an epithelial anion channel.  The identification of the CFTR gene in 1989 gave hopes of rapidly finding a cure for the disease, for which over 1950 mutations have been identified.  Since 2012, recent approaches have enabled the identification of small molecules targeting either the CFTR protein directly or its key processing steps, giving rise to novel promising therapeutic tools.  This review presents the current CFTR mutation classifications according to their clinical consequences and to their effect on the structure and function of the CFTR channel.  The future of CFTR-targeted treatment lies in combinatory therapies that will enable CF patients to receive a customized treatment.
CFTR
Genetics of cystic fibrosis: CFTR mutation classifications toward genotype-based CF therapies.Cystic fibrosis (CF) is an autosomal recessive disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which encodes an epithelial anion channel.  The identification of the CFTR gene in 1989 gave hopes of rapidly finding a cure for the disease, for which over 1950 mutations have been identified.  Since 2012, recent approaches have enabled the identification of small molecules targeting either the CFTR protein directly or its key processing steps, giving rise to novel promising therapeutic tools.  This review presents the current CFTR mutation classifications according to their clinical consequences and to their effect on the structure and function of the CFTR channel.  The future of CFTR-targeted treatment lies in combinatory therapies that will enable CF patients to receive a customized treatment.
CFTR
Genetics of cystic fibrosis: CFTR mutation classifications toward genotype-based CF therapies.Cystic fibrosis (CF) is an autosomal recessive disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which encodes an epithelial anion channel.  The identification of the CFTR gene in 1989 gave hopes of rapidly finding a cure for the disease, for which over 1950 mutations have been identified.  Since 2012, recent approaches have enabled the identification of small molecules targeting either the CFTR protein directly or its key processing steps, giving rise to novel promising therapeutic tools.  This review presents the current CFTR mutation classifications according to their clinical consequences and to their effect on the structure and function of the CFTR channel.  The future of CFTR-targeted treatment lies in combinatory therapies that will enable CF patients to receive a customized treatment.
CFTR