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Emerging Gene and Small Molecule Therapies for the Neurodevelopmental Disorder Angelman Syndrome
Angelman syndrome (AS) is a rare (~1:15,000) neurodevelopmental disorder characterized by severe developmental delay and intellectual disability, impaired communication skills, and a high prevalence of seizures, sleep disturbances, ataxia, motor deficits, and microcephaly. AS is caused by loss-of-fu...
| Autores principales: | , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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Springer International Publishing
2021
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8608975/ https://www.ncbi.nlm.nih.gov/pubmed/34528170 http://dx.doi.org/10.1007/s13311-021-01082-x |