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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...
| Autores principales: | , , , , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
American Society of Gene & Cell Therapy
2023
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| Materias: | |
| 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 |