Restoration of W1282X CFTR Activity by Enhanced Expression-Reference-Cited by-同舟云学术

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Restoration of W1282X CFTR Activity by Enhanced Expression

Published:2007-09 Issue:3 Volume:37 Page:347-356
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:

Rowe Steven M.,Varga Karoly,Rab Andras,Bebok Zsuzsa,Byram Kevin,Li Yao,Sorscher Eric J.,Clancy John P.

Publisher

American Thoracic Society

Subject

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

Reference48 articles.

1. Cystic Fibrosis

2. Correlation between Genotype and Phenotype in Patients with Cystic Fibrosis

3. Similar levels of mRNA from the W1282X and the delta F508 cystic fibrosis alleles, in nasal epithelial cells.

4. Nonsense-mediated mRNA decayin health and disease

5. Genotype-phenotype correlations in cystic fibrosis

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1. Upregulation of a nonsense mediated decay (NMD) insensitive CFTR mRNA isoform has therapeutic potential for the treatment of 3’ CFTR PTC variants;2024-07-03

2. Repair of CRISPR-guided RNA breaks enables site-specific RNA excision in human cells;Science;2024-05-17

3. Q1291H-CFTR molecular dynamics simulations and ex vivo theratyping in nasal epithelial models and clinical response to elexacaftor/tezacaftor/ivacaftor in a Q1291H/F508del patient;Frontiers in Molecular Biosciences;2023-06-01

4. The synthetic aminoglycoside ELX-02 induces readthrough of G550X-CFTR producing superfunctional protein that can be further enhanced by CFTR modulators;American Journal of Physiology-Lung Cellular and Molecular Physiology;2023-06-01

5. Gene, RNA, and ASO-based therapeutic approaches in Cystic Fibrosis;Journal of Cystic Fibrosis;2023-03

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