Investigating REPAIRv2 as a Tool to Edit <i>CFTR</i> mRNA with Premature Stop Codons

Investigating REPAIRv2 as a Tool to Edit <i>CFTR</i> mRNA with Premature Stop Codons

Cystic fibrosis (CF) is caused by mutations in the gene encoding the transmembrane conductance regulator (CFTR) protein. Some CF patients are compound heterozygous or homozygous for nonsense mutations in the <i>CFTR</i> gene. This implies the presence in the transcript of premature termi...

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Bibliographic Details
Main Authors: Raffaella Melfi, Patrizia Cancemi, Roberta Chiavetta, Viviana Barra, Laura Lentini, Aldo Di Leonardo
Format: Article
Language:English
Published: MDPI AG 2020-07-01
Series:International Journal of Molecular Sciences
Subjects:
cystic fibrosis
premature termination codons (PTCs)
RNA editing
CRISPR/dCas13b
Online Access:https://www.mdpi.com/1422-0067/21/13/4781

Internet

https://www.mdpi.com/1422-0067/21/13/4781

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