Nonsense-mediated RNA Decay Pathway Inhibition Restores Expression and Function of W1282X CFTR-Reference-Cited by-同舟云学术

Nonsense-mediated RNA Decay Pathway Inhibition Restores Expression and Function of W1282X CFTR

Published:2019-09 Issue:3 Volume:61 Page:290-300
ISSN:1044-1549
Container-title:American Journal of Respiratory Cell and Molecular Biology
language:en
Short-container-title:Am J Respir Cell Mol Biol

Author:

Keenan Melissa M.¹,Huang Lulu¹,Jordan Nikole J.²,Wong Eric²,Cheng Yi²,Valley Hillary C.²,Mahiou Jerome²,Liang Feng²,Bihler Hermann²,Mense Martin²,Guo Shuling¹,Monia Brett P.¹

Affiliation:

1. Ionis Pharmaceuticals, Carlsbad, California; and

2. Cystic Fibrosis Foundation Therapeutics Lab, Cystic Fibrosis Foundation, Lexington, Massachusetts

Publisher

American Thoracic Society

Subject

Cell Biology,Clinical Biochemistry,Pulmonary and Respiratory Medicine,Molecular Biology

Link

https://www.atsjournals.org/doi/pdf/10.1165/rcmb.2018-0316OC

Reference46 articles.

1. CFTR nonsense mutations: Therapeutic benefits from clinically approved drugs?

2. The relative frequency of CFTR mutation classes in European patients with cystic fibrosis

3. New pharmacological approaches for cystic fibrosis: Promises, progress, pitfalls

4. Restoration of W1282X CFTR Activity by Enhanced Expression

5. Synthetic Aminoglycosides Efficiently Suppress Cystic Fibrosis Transmembrane Conductance Regulator Nonsense Mutations and Are Enhanced by Ivacaftor

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