Investigating REPAIRv2 as a Tool to Edit CFTR 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 CFTR gene. This implies the presence in the transcript of premature termination codons (PTCs...

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Detalles Bibliográficos
Autores principales: Melfi, Raffaella, Cancemi, Patrizia, Chiavetta, Roberta, Barra, Viviana, Lentini, Laura, Di Leonardo, Aldo
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2020
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7369808/
https://www.ncbi.nlm.nih.gov/pubmed/32640650
http://dx.doi.org/10.3390/ijms21134781

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7369808/
https://www.ncbi.nlm.nih.gov/pubmed/32640650
http://dx.doi.org/10.3390/ijms21134781

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