Gys1 Antisense Therapy Prevents Disease-Driving Aggregates and Epileptiform Discharges in a Lafora Disease Mouse Model
Author:
Funder
Ionis Pharmaceuticals
Publisher
Elsevier BV
Subject
Pharmacology (medical),Neurology (clinical),Pharmacology
Link
https://link.springer.com/content/pdf/10.1007/s13311-023-01434-9.pdf
Reference82 articles.
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3. A novel protein tyrosine phosphatase gene is mutated in progressive myoclonus epilepsy of the Lafora type (EPM2);Serratosa;Hum Mol Genet,1999
4. Targeted disruption of the Epm2a gene causes formation of Lafora inclusion bodies, neurodegeneration, ataxia, myoclonus epilepsy and impaired behavioral response in mice;Ganesh;Hum Mol Genet,2002
5. Mutations in NHLRC1 cause progressive myoclonus epilepsy;Chan;Nat Genet,2003
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