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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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Détails bibliographiques
Publié dans:J Clin Invest
Auteurs principaux: Li, Jay, Levin, Daniel S., Kim, Audrey J., Pappas, Samuel S., Dauer, William T.
Format: Artigo
Langue:Inglês
Publié: American Society for Clinical Investigation 2021
Sujets:
Accès en ligne: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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