Literature DB >> 26046070

The holy grail of cystic fibrosis research: pharmacological repair of the F508del-CFTR mutation.

Luigi Maiuri1, Daniela De Stefano1, Valeria Raia1, Guido Kroemer1.   

Abstract

Entities:  

Year:  2015        PMID： 26046070      PMCID： PMC4437962          DOI： 10.3978/j.issn.2305-5839.2015.02.32

Source DB:  PubMed          Journal:  Ann Transl Med        ISSN： 2305-5839


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  20 in total

Review 1.  CFTR: folding, misfolding and correcting the ΔF508 conformational defect.

Authors:  Gergely L Lukacs; A S Verkman
Journal:  Trends Mol Med       Date:  2011-12-03       Impact factor: 11.951

2.  Targeting autophagy as a novel strategy for facilitating the therapeutic action of potentiators on ΔF508 cystic fibrosis transmembrane conductance regulator.

Authors:  Alessandro Luciani; Valeria Rachela Villella; Speranza Esposito; Manuela Gavina; Ilaria Russo; Marco Silano; Stefano Guido; Massimo Pettoello-Mantovani; Rosa Carnuccio; Bob Scholte; Antonella De Matteis; Maria Chiara Maiuri; Valeria Raia; Alberto Luini; Guido Kroemer; Luigi Maiuri
Journal:  Autophagy       Date:  2012-08-09       Impact factor: 16.016

3.  CFTR is a mechanosensitive anion channel: a real stretch?

Authors:  Michael A Gray
Journal:  Cellscience       Date:  2010-01

Review 4.  Pharmacologic therapy for stop mutations: how much CFTR activity is enough?

Authors:  Eitan Kerem
Journal:  Curr Opin Pulm Med       Date:  2004-11       Impact factor: 3.155

5.  Intestinal CFTR expression alleviates meconium ileus in cystic fibrosis pigs.

Authors:  David A Stoltz; Tatiana Rokhlina; Sarah E Ernst; Alejandro A Pezzulo; Lynda S Ostedgaard; Philip H Karp; Melissa S Samuel; Leah R Reznikov; Michael V Rector; Nicholas D Gansemer; Drake C Bouzek; Mahmoud H Abou Alaiwa; Mark J Hoegger; Paula S Ludwig; Peter J Taft; Tanner J Wallen; Christine Wohlford-Lenane; James D McMenimen; Jeng-Haur Chen; Katrina L Bogan; Ryan J Adam; Emma E Hornick; George A Nelson; Eric A Hoffman; Eugene H Chang; Joseph Zabner; Paul B McCray; Randall S Prather; David K Meyerholz; Michael J Welsh
Journal:  J Clin Invest       Date:  2013-05-08       Impact factor: 14.808

Review 6.  Rescuing mutant CFTR: a multi-task approach to a better outcome in treating cystic fibrosis.

Authors:  Margarida D Amaral; Carlos M Farinha
Journal:  Curr Pharm Des       Date:  2013       Impact factor: 3.116

7.  Potentiator ivacaftor abrogates pharmacological correction of ΔF508 CFTR in cystic fibrosis.

Authors:  Deborah M Cholon; Nancy L Quinney; M Leslie Fulcher; Charles R Esther; Jhuma Das; Nikolay V Dokholyan; Scott H Randell; Richard C Boucher; Martina Gentzsch
Journal:  Sci Transl Med       Date:  2014-07-23       Impact factor: 17.956

8.  Some gating potentiators, including VX-770, diminish ΔF508-CFTR functional expression.

Authors:  Guido Veit; Radu G Avramescu; Doranda Perdomo; Puay-Wah Phuan; Miklos Bagdany; Pirjo M Apaja; Florence Borot; Daniel Szollosi; Yu-Sheng Wu; Walter E Finkbeiner; Tamas Hegedus; Alan S Verkman; Gergely L Lukacs
Journal:  Sci Transl Med       Date:  2014-07-23       Impact factor: 17.956

9.  Five percent of normal cystic fibrosis transmembrane conductance regulator mRNA ameliorates the severity of pulmonary disease in cystic fibrosis.

Authors:  Anabela S Ramalho; Sebastian Beck; Michelle Meyer; Deborah Penque; Garry R Cutting; Margarida D Amaral
Journal:  Am J Respir Cell Mol Biol       Date:  2002-11       Impact factor: 6.914

10.  Restoration of CFTR function in patients with cystic fibrosis carrying the F508del-CFTR mutation.

Authors:  Daniela De Stefano; Valeria R Villella; Speranza Esposito; Antonella Tosco; Angela Sepe; Fabiola De Gregorio; Laura Salvadori; Rosa Grassia; Carlo A Leone; Giuseppe De Rosa; Maria C Maiuri; Massimo Pettoello-Mantovani; Stefano Guido; Anna Bossi; Anna Zolin; Andrea Venerando; Lorenzo A Pinna; Anil Mehta; Gianni Bona; Guido Kroemer; Luigi Maiuri; Valeria Raia
Journal:  Autophagy       Date:  2014       Impact factor: 16.016
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  2 in total

1.  Efficacy of lumacaftor-ivacaftor for the treatment of cystic fibrosis patients homozygous for the F508del-CFTR mutation.

Authors:  Deborah M Cholon; Charles R Esther; Martina Gentzsch
Journal:  Expert Rev Precis Med Drug Dev       Date:  2016-04-22

2.  A Breathe in Cystic Fibrosis Therapy: A New Therapeutic Endeavor for Cysteamine.

Authors:  Daniela De Stefano; Maria Chiara Maiuri
Journal:  EBioMedicine       Date:  2015-09-25       Impact factor: 8.143
  2 in total

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