Functional Restoration of CFTR Nonsense Mutations in Intestinal Organoids-Reference-Cited by-同舟云学术

Functional Restoration of CFTR Nonsense Mutations in Intestinal Organoids

Published:2021-10 Issue: Volume: Page:
ISSN:1569-1993
Container-title:Journal of Cystic Fibrosis
language:en
Short-container-title:Journal of Cystic Fibrosis

Author:

de Poel E.,Spelier S.,Suen S.W.F.,Kruisselbrink E.,Graeber S.Y.^ORCID,Mall M.A.^ORCID,Weersink E.J.M.^ORCID,van der Eerden M.M.,Koppelman G.H.,van der Ent C.K.,Beekman J.M.

Publisher

Elsevier BV

Subject

Pulmonary and Respiratory Medicine,Pediatrics, Perinatology and Child Health

Reference30 articles.

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2. Aminoglycoside antibiotics restore CFTR function by overcoming premature stop mutations;Howard;Nat. Med.,1996

3. PTC124 targets genetic disorders caused by nonsense mutations;Welch;Nature,2007

4. Limited premature termination codon suppression by read-through agents in cystic fibrosis intestinal organoids;Ommen;J. Cyst. Fibros.,2016

5. A lack of premature termination codon read-through efficacy of PTC124 (Ataluren) in a diverse array of reporter assays;McElroy;PLoS Biol.,2013

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3. Readthrough Activators and Nonsense-Mediated mRNA Decay Inhibitor Molecules: Real Potential in Many Genetic Diseases Harboring Premature Termination Codons;Pharmaceuticals;2024-02-28

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5. Organoid-guided synergistic treatment of minimal function CFTR mutations with CFTR modulators, roflumilast and simvastatin: a personalised approach;European Respiratory Journal;2023-10-19