Site-Specific RNA Editing of Stop Mutations in the CFTR mRNA of Human Bronchial Cultured Cells

Site-Specific RNA Editing of Stop Mutations in the CFTR mRNA of Human Bronchial Cultured Cells

It is reported that about 10% of cystic fibrosis (CF) patients worldwide have nonsense (stop) mutations in the CFTR gene, which cause the premature termination of CFTR protein synthesis, leading to a truncated and non-functional protein. To address this issue, we investigated the possibility of resc...

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Bibliographic Details
Main Authors: Roberta F. Chiavetta, Simona Titoli, Viviana Barra, Patrizia Cancemi, Raffaella Melfi, Aldo Di Leonardo
Format: Article
Language:English
Published: MDPI AG 2023-06-01
Series:International Journal of Molecular Sciences
Subjects:
cystic fibrosis
premature termination codons (PTCs)
RNA editing
CRISPR/dCas13
REPAIRv2
mxABE
Online Access:https://www.mdpi.com/1422-0067/24/13/10940

Internet

https://www.mdpi.com/1422-0067/24/13/10940

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