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

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

Abstract 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 demons...

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Bibliographic Details
Main Authors: Hyeong-Min Lee, Ellen P. Clark, M. Bram Kuijer, Mark Cushman, Yves Pommier, Benjamin D. Philpot
Format: Article
Language:English
Published: BMC 2018-08-01
Series:Molecular Autism
Subjects:
Angelman syndrome
UBE3A
Topoisomerase I
Topotecan
Indenoisoquinoline
Topoisomerase inhibitor
Online Access:http://link.springer.com/article/10.1186/s13229-018-0228-2

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http://link.springer.com/article/10.1186/s13229-018-0228-2

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