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Article

Investigating REPAIRv2 as a Tool to Edit CFTR mRNA with Premature Stop Codons.

International journal of molecular sciences - 6 Jul 2020

Melfi Raffaella, Cancemi Patrizia, Chiavetta Roberta, Barra Viviana, Lentini Laura, Di Leonardo Aldo

Abstract excerpt

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 CFTR gene. This implies the presence in the transcript of premature termination codons (PTCs) responsible for a truncated CFTR protein and a more severe form of the disease. Aminoglycoside and PTC124...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 32640650
DOI: 10.3390/ijms21134781
PMCID: PMC7369808

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