A human Angelman Syndrome class II pluripotent stem cell line with fluorescent paternal UBE3A reporter
This human-cell-based model can be used to screen novel therapeutic candidates, facilitate tracking of <i>UBE3A</i> expression in time and space, and study human-specific responses. However, its ability to restore UBE3A function cannot be studied using this model. Further research in human cells is needed to engineer systems with functional UBE3A to fully capture the therapeutic capabilities of novel candidates.
Authors
- Z. Begum Yagci (ORCID: https://orcid.org/0000-0002-1239-8680)
- Jessica K. Hartman (ORCID: https://orcid.org/0000-0003-1578-3389)
- Dilara Sen
- Linna Han (ORCID: https://orcid.org/0000-0003-3356-1763)
- Albert J. Keung
- Samantha R. Stuppy (ORCID: https://orcid.org/0000-0002-2652-0384)
- Gautami R Kelkar
- Lexi Land
Institutions
- North Carolina State University (US)
- Cell Microsystems (United States) (US)
Publication Details
- Journal
- Frontiers in Cell and Developmental Biology
- Published
- 2025-08-29
- DOI
- https://doi.org/10.3389/fcell.2025.1665693
- Citations
- 1
- Primary Topic
- Genetic Syndromes and Imprinting
- Type
- article
- Field-Weighted Citation Impact
- 1.82
Funders
- Simons Foundation Autism Research Initiative