Preliminary perspectives on gene therapy in fragile X syndrome: a caregiver view
Overall, caregivers felt hopeful, excited and interested in the prospect of gene therapy potentially providing a new treatment option, but there was some trepidation about the potential effects. Taking caregiver views into account will help inform decisions around the development and testing of any future genetic interventions.
Authors
- Sarah Eley (ORCID: https://orcid.org/0000-0001-8455-3670)
- Andrew C. Stanfield (ORCID: https://orcid.org/0000-0003-2459-1434)
- Sydni Weissgold
Institutions
- University of Edinburgh (GB)
Publication Details
- Journal
- Journal of Neurodevelopmental Disorders
- Published
- 2025-09-01
- DOI
- https://doi.org/10.1186/s11689-025-09629-1
- Primary Topic
- Genetics and Neurodevelopmental Disorders
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- FRAXA Research Foundation