Transduction of hematopoietic stem and progenitor cells by an MECP2 lentiviral vector improves Rett syndrome phenotypes
By providing functional MeCP2 to affected cells, our results highlight the ability of this strategy to improve Rett syndrome phenotypes. These proof-of-concept studies demonstrate the potential use of a stem cell gene therapy approach as a novel treatment for Rett syndrome patients.
Authors
- Joseph S. Anderson (ORCID: https://orcid.org/0000-0001-6001-1156)
- Jill L. Silverman (ORCID: https://orcid.org/0000-0001-9357-5476)
- Julie R. Beegle
- Alyse L. Lodigiani (ORCID: https://orcid.org/0009-0009-7744-7138)
- Camilla M. Barbaduomo
Institutions
- University of California Davis Medical Center (US)
Publication Details
- Journal
- Frontiers in Drug Discovery
- Published
- 2025-02-21
- DOI
- https://doi.org/10.3389/fddsv.2025.1545391
- Primary Topic
- Genetics and Neurodevelopmental Disorders
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Rett Syndrome Research Trust