Heet Naresh Kaku

Jan 24, 2025 | digitalcommons.library.tmc.edu | Dongwon Lee |Wu Chen |Heet Naresh Kaku |Xinming Zhuo

UBE3A encodes ubiquitin protein ligase E3A, and in neurons its expression from the paternal allele is repressed by the UBE3A antisense transcript (UBE3A-ATS). This leaves neurons susceptible to loss-of-function of maternal UBE3A. Indeed, Angelman syndrome, a severe neurodevelopmental disorder, is caused by maternal UBE3A deficiency. A promising therapeutic approach to treating Angelman syndrome is to reactivate the intact paternal UBE3A by suppressing UBE3A-ATS.

Jul 3, 2023 | biorxiv.org | Amanda Brown |Heet Naresh Kaku |Roy V. Sillitoe

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