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The prospect of molecular therapy for Angelman syndrome and other monogenic neurologic disorders

BACKGROUND: Angelman syndrome is a monogenic neurologic disorder that affects 1 in 15,000 children, and is characterized by ataxia, intellectual disability, speech impairment, sleep disorders, and seizures. The disorder is caused by loss of central nervous system expression of UBE3A, a gene encoding...

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Detalles Bibliográficos
Autores principales: Bailus, Barbara J, Segal, David J
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2014
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4069279/
https://www.ncbi.nlm.nih.gov/pubmed/24946931
http://dx.doi.org/10.1186/1471-2202-15-76