Pre‐clinical development of AVB‐101, an AAV gene therapy treatment for frontotemporal dementia with progranulin mutations
The preclinical data suggest that intrathalamic infusion of AVB-101 constitutes a novel and promising approach to supplement PGRN in the CNS, supporting the clinical development of AVB-101 for FTD-GRN.
Authors
- Joseph Allison (ORCID: https://orcid.org/0000-0002-0638-7678)
- Page Bouchard (ORCID: https://orcid.org/0000-0002-5528-1941)
- A. Joseph Bloom (ORCID: https://orcid.org/0000-0002-6731-5539)
- Olivier Brock (ORCID: https://orcid.org/0000-0003-1965-7531)
- Romain Joubert (ORCID: https://orcid.org/0000-0003-4902-2035)
- Carlos J. Miranda (ORCID: https://orcid.org/0000-0001-8208-7100)
- Aleksandra Kaliszewska (ORCID: https://orcid.org/0000-0002-6458-0409)
- McQuinn Gumus
- Alinda R. Fernandes
- Youn‐Bok Lee (ORCID: https://orcid.org/0000-0002-0479-9361)
- Christopher E. Shaw (ORCID: https://orcid.org/0000-0003-0251-0146)
- Paniz Hosseini
- José Furtado (ORCID: https://orcid.org/0000-0002-7541-6305)
- Zuzana Walker (ORCID: https://orcid.org/0000-0001-7346-8200)
- Do Young Lee (ORCID: https://orcid.org/0000-0002-5868-2628)
- Yalem Bekele
Institutions
- King's College London (GB)
- Healthcentric Advisors (US)
- UK Dementia Research Institute (GB)
Publication Details
- Journal
- Alzheimer s & Dementia
- Published
- 2024-12-01
- DOI
- https://doi.org/10.1002/alz.086546
- Primary Topic
- Amyotrophic Lateral Sclerosis Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00