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Antisense oligonucleotide-based drug development for Cystic Fibrosis patients carrying the 3849 + 10 kb C-to-T splicing mutation

BACKGROUND: Antisense oligonucleotide (ASO)-based drugs for splicing modulation were recently approved for various genetic diseases with unmet need. Here we aimed to develop an ASO-based splicing modulation therapy for Cystic Fibrosis (CF) patients carrying the 3849 + 10 kb C-to-T splicing mutation...

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Detalles Bibliográficos
Autores principales:	Oren, Yifat S., Irony-Tur Sinai, Michal, Golec, Anita, Barchad-Avitzur, Ofra, Mutyam, Venkateshwar, Li, Yao, Hong, Jeong, Ozeri-Galai, Efrat, Hatton, Aurélie, Leibson, Chen, Carmel, Liran, Reiter, Joel, Sorscher, Eric J., Wilton, Steve D., Kerem, Eitan, Rowe, Steven M., Sermet-Gaudelus, Isabelle, Kerem, Batsheva
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	2021
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8464507/ https://www.ncbi.nlm.nih.gov/pubmed/34226157 http://dx.doi.org/10.1016/j.jcf.2021.06.003

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