Literature DB >> 31978337

A Therapy for Most with Cystic Fibrosis.

Christine E Bear1.   

Abstract

TRIKAFTA is the third drug approved by the FDA that rescues defects caused by the major mutation F508del. It is superior to its predecessors that were approved for patients who are homozygous for F508del because TRIKAFTA is also effective in CF patients who harbor only one copy of this mutation.
Copyright © 2019. Published by Elsevier Inc.

Entities:  

Year:  2020        PMID： 31978337     DOI： 10.1016/j.cell.2019.12.032

Source DB:  PubMed          Journal:  Cell        ISSN： 0092-8674            Impact factor:   41.582


  15 in total

1.  Elexacaftor-Tezacaftor-Ivacaftor as a Final Frontier in the Treatment of Cystic Fibrosis: Definition of the Clinical and Microbiological Implications in a Case-Control Study.

Authors:  Giuseppe Migliorisi; Mirella Collura; Francesca Ficili; Tiziana Pensabene; Dafne Bongiorno; Antonina Collura; Francesca Di Bernardo; Stefania Stefani
Journal:  Pharmaceuticals (Basel)       Date:  2022-05-14

Review 2.  CFTR and Gastrointestinal Cancers: An Update.

Authors:  Rahul Bhattacharya; Zachary Blankenheim; Patricia M Scott; Robert T Cormier
Journal:  J Pers Med       Date:  2022-05-25

3.  A restructuring of microbiome niche space is associated with Elexacaftor-Tezacaftor-Ivacaftor therapy in the cystic fibrosis lung.

Authors:  Lo M Sosinski; Christian Martin H; Kerri A Neugebauer; Lydia-Ann J Ghuneim; Douglas V Guzior; Alicia Castillo-Bahena; Jenna Mielke; Ryan Thomas; Marc McClelland; Doug Conrad; Robert A Quinn
Journal:  J Cyst Fibros       Date:  2021-11-22       Impact factor: 5.527

4.  Enhanced delivery of peptide-morpholino oligonucleotides with a small molecule to correct splicing defects in the lung.

Authors:  Yan Dang; Catharina van Heusden; Veronica Nickerson; Felicity Chung; Yang Wang; Nancy L Quinney; Martina Gentzsch; Scott H Randell; Hong M Moulton; Ryszard Kole; Aiguo Ni; Rudolph L Juliano; Silvia M Kreda
Journal:  Nucleic Acids Res       Date:  2021-06-21       Impact factor: 16.971

Review 5.  Human Molecular Genetics and the long road to treating cystic fibrosis.

Authors:  Ann Harris
Journal:  Hum Mol Genet       Date:  2021-10-01       Impact factor: 5.121

Review 6.  New Directions in Pulmonary Gene Therapy.

Authors:  Amber Vu; Paul B McCray
Journal:  Hum Gene Ther       Date:  2020-09       Impact factor: 4.793

7.  Quantitative Evaluation of CFTR Pre-mRNA Splicing Dependent on the (TG)mTn Poly-Variant Tract.

Authors:  Manuela Sterrantino; Andrea Fuso; Silvia Pierandrei; Sabina Maria Bruno; Giancarlo Testino; Giuseppe Cimino; Antonio Angeloni; Marco Lucarelli
Journal:  Diagnostics (Basel)       Date:  2021-01-25

Review 8.  A Review of Trikafta: Triple Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Modulator Therapy.

Authors:  Anas Zaher; Jude ElSaygh; Dalal Elsori; Hassan ElSaygh; Abdulsabar Sanni
Journal:  Cureus       Date:  2021-07-03

Review 9.  Cystic Fibrosis Human Organs-on-a-Chip.

Authors:  Herbert Luke Ogden; Hoyeol Kim; Kathryn A Wikenheiser-Brokamp; Anjaparavanda P Naren; Kyu Shik Mun
Journal:  Micromachines (Basel)       Date:  2021-06-25       Impact factor: 2.891

Review 10.  Immunomodulation in Cystic Fibrosis: Why and How?

Authors:  Vincent D Giacalone; Brian S Dobosh; Amit Gaggar; Rabindra Tirouvanziam; Camilla Margaroli
Journal:  Int J Mol Sci       Date:  2020-05-08       Impact factor: 5.923
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