Targeted exon skipping of NF1 exon 17 as a therapeutic for neurofibromatosis type I

We investigated the feasibility of utilizing an exon-skipping approach as a genotype-dependent therapeutic for neurofibromatosis type 1 (NF1) by determining which NF1 exons might be skipped while maintaining neurofibromin protein expression and GTPase activating protein (GAP)-related domain (GRD) fu...

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Detalles Bibliográficos
Autores principales: Leier, André, Moore, Marc, Liu, Hui, Daniel, Michael, Hyde, Alexis M., Messiaen, Ludwine, Korf, Bruce R., Selvakumaran, Jamuna, Ciszewski, Lukasz, Lambert, Laura, Foote, Jeremy, Wallace, Margaret R., Kesterson, Robert A., Dickson, George, Popplewell, Linda, Wallis, Deeann
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8983316/
https://www.ncbi.nlm.nih.gov/pubmed/35433111
http://dx.doi.org/10.1016/j.omtn.2022.03.011

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8983316/
https://www.ncbi.nlm.nih.gov/pubmed/35433111
http://dx.doi.org/10.1016/j.omtn.2022.03.011

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