CRISPR/Cas9 directed to the Ube3a antisense transcript improves Angelman syndrome phenotype in mice.
Gene editing holds the potential to correct mutations and cure devastating genetic disorders. The technology has not yet proven efficacious for therapeutic use in CNS diseases with ubiquitous neuronal defects. Angelman syndrome (AS), a severe neurodevelopmental disorder, is caused by a lack of mater...
| Publicado en: | Journal of Clinical Investigation Vol. 131; no. 5; pp. 1 - 8 |
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| Autores principales: | , , , , , , |
| Formato: | research Journal Article |
| Publicado: |
American Society for Clinical Investigation
Mar2021
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| Acceso en línea: | Ver este registro en EBSCOhost |