Genetics of Cystic Fibrosis: Clinical Implications.
暂无分享,去创建一个
[1] J. Davies,et al. The future of CFTR modulating therapies for cystic fibrosis , 2015, Current opinion in pulmonary medicine.
[2] P. Davis. Another Beginning for Cystic Fibrosis Therapy. , 2015, The New England journal of medicine.
[3] Xiaohong Huang,et al. Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR. , 2015, The New England journal of medicine.
[4] A. Nicholson,et al. Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial , 2015, The Lancet. Respiratory medicine.
[5] S. McColley,et al. Efficacy and safety of ivacaftor in patients with cystic fibrosis who have an Arg117His-CFTR mutation: a double-blind, randomised controlled trial. , 2015, The Lancet. Respiratory medicine.
[6] E. Alton,et al. Cystic Fibrosis Gene Therapy in the UK and Elsewhere , 2015, Human gene therapy.
[7] D. Sheppard,et al. Chronic ivacaftor treatment: getting F508del-CFTR into more trouble? , 2014, Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society.
[8] Garry R. Cutting,et al. Cystic fibrosis genetics: from molecular understanding to clinical application , 2014, Nature Reviews Genetics.
[9] Ying Jiang,et al. Long-term safety and efficacy of ivacaftor in patients with cystic fibrosis who have the Gly551Asp-CFTR mutation: a phase 3, open-label extension study (PERSIST). , 2014, The Lancet. Respiratory medicine.
[10] N. Dokholyan,et al. Potentiator ivacaftor abrogates pharmacological correction of ΔF508 CFTR in cystic fibrosis , 2014, Science Translational Medicine.
[11] G. Lukács,et al. Some gating potentiators, including VX-770, diminish ΔF508-CFTR functional expression , 2014, Science Translational Medicine.
[12] S. McColley,et al. A CFTR corrector (lumacaftor) and a CFTR potentiator (ivacaftor) for treatment of patients with cystic fibrosis who have a phe508del CFTR mutation: a phase 2 randomised controlled trial. , 2014, The Lancet. Respiratory medicine.
[13] S. Peltz,et al. Ataluren for the treatment of nonsense-mutation cystic fibrosis: a randomised, double-blind, placebo-controlled phase 3 trial. , 2014, The Lancet. Respiratory medicine.
[14] S. Bicknell,et al. Effects of ivacaftor in patients with cystic fibrosis who carry the G551D mutation and have severe lung disease. , 2014, Chest.
[15] Chris Fellner,et al. CFTR Modulators for the Treatment of Cystic Fibrosis. , 2014, P & T : a peer-reviewed journal for formulary management.
[16] Hans Clevers,et al. Functional repair of CFTR by CRISPR/Cas9 in intestinal stem cell organoids of cystic fibrosis patients. , 2013, Cell stem cell.
[17] Marisa Sousa,et al. Revertants, low temperature, and correctors reveal the mechanism of F508del-CFTR rescue by VX-809 and suggest multiple agents for full correction. , 2013, Chemistry & biology.
[18] A. Verkman,et al. Cystic fibrosis transmembrane regulator correctors and potentiators. , 2013, Cold Spring Harbor perspectives in medicine.
[19] L. Galietta. Managing the Underlying Cause of Cystic Fibrosis: A Future Role for Potentiators and Correctors , 2013, Pediatric Drugs.
[20] J. Mainz,et al. Efficacy and safety of ivacaftor in patients aged 6 to 11 years with cystic fibrosis with a G551D mutation. , 2013, American journal of respiratory and critical care medicine.
[21] M. Boyle,et al. A new era in the treatment of cystic fibrosis: correction of the underlying CFTR defect. , 2013, The Lancet. Respiratory medicine.
[22] I. Sermet-Gaudelus. Ivacaftor treatment in patients with cystic fibrosis and the G551D-CFTR mutation , 2013, European Respiratory Review.
[23] D. Gell,et al. Hemoglobin variants: biochemical properties and clinical correlates. , 2013, Cold Spring Harbor perspectives in medicine.
[24] M. Wilschanski. Novel therapeutic approaches for cystic fibrosis. , 2013, Discovery medicine.
[25] T. Liou,et al. Ivacaftor in subjects with cystic fibrosis who are homozygous for the F508del-CFTR mutation. , 2012, Chest.
[26] Matthias Griese,et al. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. , 2011, The New England journal of medicine.
[27] P. Negulescu,et al. Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809 , 2011, Proceedings of the National Academy of Sciences.
[28] J. Clancy,et al. Results of a phase IIa study of VX-809, an investigational CFTR corrector compound, in subjects with cystic fibrosis homozygous for the F508del-CFTR mutation , 2011, Thorax.
[29] H. Blau,et al. Chronic ataluren (PTC124) treatment of nonsense mutation cystic fibrosis , 2011, European Respiratory Journal.
[30] J. Gustafson,et al. Cystic Fibrosis , 2009, Journal of the Iowa Medical Society.
[31] M. Bibikova,et al. Efficient Gene Targeting in Drosophila With Zinc-Finger Nucleases , 2006, Genetics.
[32] G. Novelli,et al. Isolation of CF cell lines corrected at ΔF508-CFTR locus by SFHR-mediated targeting , 2002, Gene Therapy.
[33] B. Dallapiccola,et al. Expression of ΔF508 CFTR in normal mouse lung after site-specific modification of CFTR sequences by SFHR , 2001, Gene Therapy.
[34] K. Kunzelmann,et al. Targeted replacement of normal and mutant CFTR sequences in human airway epithelial cells using DNA fragments. , 1998, Human molecular genetics.
[35] S. Magness,et al. Distinct levels of Sox9 expression mark colon epithelial stem cells that form colonoids in culture. , 2012, American journal of physiology. Gastrointestinal and liver physiology.