Characterization and structure-activity relationships of indenoisoquinoline-derived topoisomerase I inhibitors in unsilencing the dormant Ube3a gene associated with Angelman syndrome

BACKGROUND: Angelman syndrome (AS) is a severe neurodevelopmental disorder lacking effective therapies. AS is caused by mutations in ubiquitin protein ligase E3A (UBE3A), which is genomically imprinted such that only the maternally inherited copy is expressed in neurons. We previously demonstrated t...

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Bibliografische gegevens
Gepubliceerd in:Mol Autism
Hoofdauteurs: Lee, Hyeong-Min, Clark, Ellen P., Kuijer, M. Bram, Cushman, Mark, Pommier, Yves, Philpot, Benjamin D.
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: BioMed Central 2018
Onderwerpen:
Research
Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6098585/
https://ncbi.nlm.nih.gov/pubmed/30140420
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13229-018-0228-2
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