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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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Gorde:
Xehetasun bibliografikoak
Argitaratua izan da:J Clin Invest
Egile Nagusiak: Li, Jay, Levin, Daniel S., Kim, Audrey J., Pappas, Samuel S., Dauer, William T.
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: American Society for Clinical Investigation 2021
Gaiak:
Sarrera elektronikoa: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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