Gene therapy for presenilin mutation carriers
We provide proof of concept that it is possible to generate active ASOs selectively targeting mutant PSEN1, addressing the genetic root cause of ADAD. Such gene therapy approach could be applied to all ADAD causing mutations and potentially addressing the disease before symptoms appear, delaying or even preventing the progression of pathology.
Authors
- Inmaculada Sanjuan Ruiz
Institutions
- VIB-KU Leuven Center for Brain & Disease Research (BE)
Publication Details
- Journal
- Alzheimer s & Dementia
- Published
- 2025-12-01
- DOI
- https://doi.org/10.1002/alz70859_098883
- Primary Topic
- Alzheimer's disease research and treatments
- Type
- article
- Field-Weighted Citation Impact
- 0.00