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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...
| Autores principales: | , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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BioMed Central
2014
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| 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 |