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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Detalhes bibliográficos
Publicado no:Hum Mol Genet
Main Authors: 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: Artigo
Idioma:Inglês
Publicado em: Oxford University Press 2021
Assuntos:
General Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC8188406/
https://ncbi.nlm.nih.gov/pubmed/33856035
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddab104
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