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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Detalles Bibliográficos
Autores principales: Lee, Hyeong-Min, Clark, Ellen P., Kuijer, M. Bram, Cushman, Mark, Pommier, Yves, Philpot, Benjamin D.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2018
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6098585/
https://www.ncbi.nlm.nih.gov/pubmed/30140420
http://dx.doi.org/10.1186/s13229-018-0228-2

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6098585/
https://www.ncbi.nlm.nih.gov/pubmed/30140420
http://dx.doi.org/10.1186/s13229-018-0228-2

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