Protracted CLN3 Batten disease in mice that genetically model an exon-skipping therapeutic approach

Genetic mutations that disrupt open reading frames and cause translation termination are frequent causes of human disease and are difficult to treat due to protein truncation and mRNA degradation by nonsense-mediated decay, leaving few options for traditional drug targeting. Splice-switching antisen...

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Detalles Bibliográficos
Autores principales: Centa, Jessica L., Stratton, Matthew P., Pratt, Melissa A., Osterlund Oltmanns, Jenna R., Wallace, Douglas G., Miller, Steven A., Weimer, Jill M., Hastings, Michelle L.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10285469/
https://www.ncbi.nlm.nih.gov/pubmed/37359347
http://dx.doi.org/10.1016/j.omtn.2023.05.025

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10285469/
https://www.ncbi.nlm.nih.gov/pubmed/37359347
http://dx.doi.org/10.1016/j.omtn.2023.05.025

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