Advancing Hamartin Therapy in a TSC1 Mouse Model: AAV Delivery and Enhancement with Extracellular Vesicles (P10-2.009)
To evaluate therapeutic effects of AAV-mediated hamartin delivery in a TSC1 mouse model and investigate the potential of extracellular vesicles (EVs) to enhance hamartin distribution and function, aiming to improve treatment efficacy for TSC1-associated pathologies.
Authors
- Iván Coto Hernández (ORCID: https://orcid.org/0000-0003-3611-7959)
- Vijaya Ramesh (ORCID: https://orcid.org/0000-0003-4966-5268)
- Edwina Abou Haidar (ORCID: https://orcid.org/0000-0002-7866-8030)
- Roberta L. Beauchamp
- Shilpa Prabhakar (ORCID: https://orcid.org/0000-0002-7999-2000)
- Koen Breyne (ORCID: https://orcid.org/0000-0002-0713-2407)
- Xandra O. Breakefield (ORCID: https://orcid.org/0000-0001-6036-0399)
- Tomeh Tomeh
Institutions
- Massachusetts General Hospital (US)
- Neurology, Inc (US)
Publication Details
- Journal
- Neurology
- Published
- 2025-04-07
- DOI
- https://doi.org/10.1212/wnl.0000000000212042
- Citations
- 1
- Primary Topic
- Extracellular vesicles in disease
- Type
- article
- Field-Weighted Citation Impact
- 0.81