Combining Engineered U1 snRNA and Antisense Oligonucleotides to Improve the Treatment of a BBS1 Splice Site Mutation

Manipulation of pre-mRNA processing is a promising approach toward overcoming disease-causing mutations and treating human diseases. We show that a combined treatment applying two splice-manipulating technologies improves therapeutic efficacies to correct mutation-induced splice defects. Previously,...

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Detalles Bibliográficos
Autores principales: Breuel, Saskia, Vorm, Mariann, Bräuer, Anja U., Owczarek-Lipska, Marta, Neidhardt, John
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2019
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6796732/
https://www.ncbi.nlm.nih.gov/pubmed/31541798
http://dx.doi.org/10.1016/j.omtn.2019.08.014

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6796732/
https://www.ncbi.nlm.nih.gov/pubmed/31541798
http://dx.doi.org/10.1016/j.omtn.2019.08.014

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