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Functional rescue in an Angelman syndrome model following treatment with lentivector transduced hematopoietic stem cells

Angelman syndrome (AS) is a rare neurodevelopmental disorder characterized by impaired communication skills, ataxia, motor and balance deficits, intellectual disabilities, and seizures. The genetic cause of AS is the neuronal loss of UBE3A expression in the brain. A novel approach, described here, i...

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Detalles Bibliográficos
Autores principales:	Adhikari, Anna, Copping, Nycole A, Beegle, Julie, Cameron, David L, Deng, Peter, O’Geen, Henriette, Segal, David J, Fink, Kyle D, Silverman, Jill L, Anderson, Joseph S
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Oxford University Press 2021
Materias:	General Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8188406/ https://www.ncbi.nlm.nih.gov/pubmed/33856035 http://dx.doi.org/10.1093/hmg/ddab104

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