Rescue of FTLD‐associated TDP‐43 pathology and neurodegeneration by peripheral AAV‐mediated expression of brain‐penetrant progranulin
Together, our studies suggest that peripherally administered brain-penetrant PGRN replacement strategies can ameliorate FTLD-GRN relevant phenotypes including TDP-43 pathology, neurodegeneration and behavioral deficits. Our data provide preclinical proof of concept for the use of this AAV platform for treatment of FTLD-GRN and potentially other CNS disorders.
Authors
- Christian Haass (ORCID: https://orcid.org/0000-0002-4869-1627)
Institutions
- German Center for Neurodegenerative Diseases (DE)
Publication Details
- Journal
- Alzheimer s & Dementia
- Published
- 2024-12-01
- DOI
- https://doi.org/10.1002/alz.090862
- Primary Topic
- Amyotrophic Lateral Sclerosis Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00