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TorsinA restoration in a mouse model identifies a critical therapeutic window for DYT1 dystonia
In inherited neurodevelopmental diseases, pathogenic processes unique to critical periods during early brain development may preclude the effectiveness of gene modification therapies applied later in life. We explored this question in a mouse model of DYT1 dystonia, a neurodevelopmental disease caus...
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| Опубликовано в: : | J Clin Invest |
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| Главные авторы: | , , , , |
| Формат: | Artigo |
| Язык: | Inglês |
| Опубликовано: |
American Society for Clinical Investigation
2021
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| Предметы: | |
| Online-ссылка: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7954593/ https://ncbi.nlm.nih.gov/pubmed/33529159 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/JCI139606 |
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