Novel Exon-Skipping Therapeutic Approach for the DMD Gene Based on Asymptomatic Deletions of Exon 49

Exon skipping is a promising therapeutic approach. One important condition for this approach is that the exon-skipped form of the gene can at least partially perform the required function and lead to improvement of the phenotype. It is therefore critical to identify the exons that can be skipped wit...

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Detalles Bibliográficos
Autores principales: Abaji, Mario, Gorokhova, Svetlana, Da Silva, Nathalie, Busa, Tiffany, Grelet, Maude, Missirian, Chantal, Sigaudy, Sabine, Philip, Nicole, Leturcq, France, Lévy, Nicolas, Krahn, Martin, Bartoli, Marc
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9323532/
https://www.ncbi.nlm.nih.gov/pubmed/35886062
http://dx.doi.org/10.3390/genes13071277

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9323532/
https://www.ncbi.nlm.nih.gov/pubmed/35886062
http://dx.doi.org/10.3390/genes13071277

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