RNAi-Induced Expression of Paternal UBE3A
shRNA-mediated inhibition of <i>Ube3a</i>-ATS by targeting <i>Snord115</i> effectively restores Ube3a/UBE3A expression in both mouse neurons and human iPSCs. While promising, the mild reduction in <i>Snord116</i> raises concerns about potential off-target effects. AAV-based delivery of shRNA shows potential, but its translational applicability remains to be evaluated in vivo.
Authors
- Violeta Zaric
- S. Gray
- Volodymyr Rybalchenko (ORCID: https://orcid.org/0000-0003-4092-0779)
- Hye Ri Kang
- Ryan K. Butler
Institutions
- Southwestern Medical Center (US)
- Southwestern Medical Center (US)
- The University of Texas Southwestern Medical Center (US)
Publication Details
- Journal
- Genes
- Published
- 2026-01-29
- DOI
- https://doi.org/10.3390/genes17020156
- Primary Topic
- Genetic Syndromes and Imprinting
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Angelman Syndrome Foundation