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10.1136/bmj.i859 http://scihub22266oqcxt.onion/10.1136/bmj.i859 C4817245!4817245 !27030675     free     free     free Warning: file_get_contents(https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pubmed&id=27030675 &cmd=llinks): Failed to open stream: HTTP request failed! HTTP/1.1 429 Too Many Requests in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 215 Deprecated: Implicit conversion from float 209.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 209.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Warning: imagejpeg(C:\Inetpub\vhosts\kidney.de\httpdocs\phplern\27030675 .jpg): Failed to open stream: No such file or directory in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 117       BMJ 2016 ; 352 (ä): i859 Nephropedia Template TP {{tp|p=27030675 |t=2016. New and emerging targeted therapies for cystic fibrosis |pdf=|usr=}}{{27030675 }} gab.com Text New and emerging targeted therapies for cystic fibrosis JO: BMJ http://www.kidney.de/pget.php?&tw=1&pmid=27030675 #FOAvip Cystic fibrosis (CF) is a monogenic autosomal recessive disorder that affects about 70,000 people worldwide. The clinical manifestations of the disease are caused by defects in the cystic fibrosis transmembrane conductance regulator (CFTR) protein. The discovery of the CFTR gene in 1989 has led to a sophisticated understanding of how thousands of mutations in the CFTR gene affect the structure and function of the CFTR protein. Much progress has been made over the past decade with the development of orally bioavailable small molecule drugs that target defective CFTR proteins caused by specific mutations. Furthermore, there is considerable optimism about the prospect of gene replacement or editing therapies to correct all mutations in cystic fibrosis. The recent approvals of ivacaftor and lumacaftor represent the genesis of a new era of precision medicine in the treatment of this condition. These drugs are having a positive impact on the lives of people with cystic fibrosis and are potentially disease modifying. This review provides an update on advances in our understanding of the structure and function of the CFTR, with a focus on state of the art targeted drugs that are in development. Twit Text FOAVip New and emerging targeted therapies for cystic fibrosis ????BMJ http://www.kidney.de/pget.php?&tw=1&pmid=27030675 #FOAvip Twit Text # Free Paper on # # # # # LINK http://www.kidney.de/pget.php?&tw=1&pmid=27030675 #FOAvip English Wikipedia <ref>{{Cite pmid|27030675 }}</ref> New and emerging targeted therapies for cystic fibrosis #MMPMID27030675 Quon BS ; Rowe SM BMJ 2016[Mar]; 352 (ä): i859 PMID27030675 show ga Cystic fibrosis (CF) is a monogenic autosomal recessive disorder that affects about 70,000 people worldwide. The clinical manifestations of the disease are caused by defects in the cystic fibrosis transmembrane conductance regulator (CFTR) protein. The discovery of the CFTR gene in 1989 has led to a sophisticated understanding of how thousands of mutations in the CFTR gene affect the structure and function of the CFTR protein. Much progress has been made over the past decade with the development of orally bioavailable small molecule drugs that target defective CFTR proteins caused by specific mutations. Furthermore, there is considerable optimism about the prospect of gene replacement or editing therapies to correct all mutations in cystic fibrosis. The recent approvals of ivacaftor and lumacaftor represent the genesis of a new era of precision medicine in the treatment of this condition. These drugs are having a positive impact on the lives of people with cystic fibrosis and are potentially disease modifying. This review provides an update on advances in our understanding of the structure and function of the CFTR, with a focus on state of the art targeted drugs that are in development. |Aminophenols/therapeutic use [MESH] |Aminopyridines/therapeutic use [MESH] |Benzodioxoles/therapeutic use [MESH] |Clinical Trials as Topic [MESH] |Cystic Fibrosis Transmembrane Conductance Regulator/drug effects/*genetics/physiology [MESH] |Cystic Fibrosis/genetics/*therapy [MESH] |Drug Discovery/trends [MESH] |Drug Therapy, Combination [MESH] |Forced Expiratory Volume/drug effects [MESH] |Forecasting [MESH] |Genetic Therapy/methods/trends [MESH] |Homozygote [MESH] |Humans [MESH] |Molecular Targeted Therapy/methods/trends [MESH] |Mutation/genetics [MESH] |Oxadiazoles/therapeutic use [MESH] |Phosphodiesterase 5 Inhibitors/therapeutic use [MESH] |Practice Guidelines as Topic [MESH] |Precision Medicine/methods/trends [MESH]New and emerging targeted therapies for cystic fibrosis (epmc).pdf DeepDyve Pubget Overpricing