Molecular and functional correction of a deep intronic splicing mutation in CFTR by CRISPR-Cas9 gene editing

Cystic fibrosis (CF) is an autosomal recessive disorder caused by mutations in the CFTR gene. The 10th most common mutation, c.3178-2477C>T (3849+10kb C>T), involves a cryptic, intronic splice site. This mutation was corrected in CF primary cells homozygous for this mutation by delivering pair...

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Detalles Bibliográficos
Autores principales: Walker, Amy J., Graham, Carina, Greenwood, Miriam, Woodall, Maximillian, Maeshima, Ruhina, O’Hara-Wright, Michelle, Sanz, David J., Guerrini, Ileana, Aldossary, Ahmad M., O’Callaghan, Christopher, Baines, Deborah L., Harrison, Patrick T., Hart, Stephen L.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10661860/
https://www.ncbi.nlm.nih.gov/pubmed/38027060
http://dx.doi.org/10.1016/j.omtm.2023.101140

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