Dual-isoform hUBE3A gene transfer improves behavioral and seizure outcomes in Angelman syndrome model mice

Loss of the maternal UBE3A allele causes Angelman syndrome (AS), a debilitating neurodevelopmental disorder. Here, we devised an AS treatment strategy based on reinstating dual-isoform expression of human UBE3A (hUBE3A) in the developing brain. Kozak sequence engineering of our codon-optimized vecto...

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Detalles Bibliográficos
Autores principales: Judson, Matthew C., Shyng, Charles, Simon, Jeremy M., Davis, Courtney R., Punt, A. Mattijs, Salmon, Mirabel T., Miller, Noah W., Ritola, Kimberly D., Elgersma, Ype, Amaral, David G., Gray, Steven J., Philpot, Benjamin D.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society for Clinical Investigation 2021
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8564914/
https://www.ncbi.nlm.nih.gov/pubmed/34676830
http://dx.doi.org/10.1172/jci.insight.144712

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8564914/
https://www.ncbi.nlm.nih.gov/pubmed/34676830
http://dx.doi.org/10.1172/jci.insight.144712

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