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Prenatal treatment path for Angelman syndrome and other neurodevelopmental disorders
Angelman syndrome (AS) is a rare neurodevelopmental disorder caused by mutation or deletion of the maternally-inherited UBE3A allele. These pathogenic mutations lead to loss of maternal UBE3A expression in neurons. Antisense oligonucleotides and gene therapies are in development that activate the in...
Gorde:
| Argitaratua izan da: | Autism Res |
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| Egile nagusia: | |
| Formatua: | Artigo |
| Hizkuntza: | Inglês |
| Argitaratua: |
2019
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| Gaiak: | |
| Sarrera elektronikoa: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7968581/ https://ncbi.nlm.nih.gov/pubmed/31490639 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/aur.2203 |
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