Recent advancements in treatment of cystic fibrosis
Sumit Devkar, Piyush Jangam, Yogesh bafana, Pratik Jadhav, Shubham Ghorpade, Shraddha Bhor, Swamini Kute
CFTR modulators, Ivacaftor, Mucociliary clearance, Physiology, PTC124
Cystic fibrosis (CF) is the most usual life-shortening autosomal recessive disease(1). It is generally result of mutations in the CFTR (cystic fibrosis transmembrane conductance regulator) gene (2) . This result into the accumulation of exocrine secretions in various systems, specifically the respiratory, and gastrointestinal systems, causing serious comorbidities(5). Still, lung disorder accounts for important reason for the excessive morbidity and mortality in CF(6)Cystic fibrosis also influences epithelial cell lining of reproductive tract and lead to faulty exocrine secretion.Patients with cystic fibrosis have regular gastrointestinal issues like Fatty liver , Rectal prolapse , Constipation , malabsorption , Distal intestinal obstruction syndrome, Gastro-oesophageal reflux , etc(3). Most general CF-causing mutation is F508del. Other CFTR mutations may additionally impair mRNA and protein expression, function, stability or a combination of these. Gene modifiers, social status, patient's lifestyle, respiratory infections and adherence to treatment plans like quite a few different elements impact disorder severity beyond CFTR mutations. Most of the new drugs availablee in market for CF remedy have been deliberate and developed from acquired advances on genetics and proteomics(6) Kalydeco® (Ivacaftor),is a drug referred to as a CFTR “potentiator” was at the beginning supposed for use in the therapy of patients with the G551D mutation in Cystic Fibrosis(7).Research is being carried out into gene therapy that aims at introducing a normal copy of CFTR into lung epithelial cells.
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Unique Paper ID: 160674

Publication Volume & Issue: Volume 10, Issue 1

Page(s): 978 - 987
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