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Genome Editing for Cystic Fibrosis

Cystic fibrosis (CF) is a monogenic recessive genetic disorder caused by mutations in the CF Transmembrane-conductance Regulator gene (CFTR). Remarkable progress in basic research has led to the discovery of highly effective CFTR modulators. Now ~90% of CF patients are treatable. However, these modu...

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Detalles Bibliográficos
Autor principal: Wang, Guoshun
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10297084/
https://www.ncbi.nlm.nih.gov/pubmed/37371025
http://dx.doi.org/10.3390/cells12121555