Humanized mice carrying a pathogenic GRN deletion as a pre-clinical platform for targeted gene therapies in frontotemporal dementia
model provides insight into progranulin biology, increases our understanding of a pathogenic variant that causes FTD, and facilitates the development of GRN gene therapies.
Authors
- Keegan Korthauer (ORCID: https://orcid.org/0000-0002-4565-1654)
- Sarah B. Thomson (ORCID: https://orcid.org/0000-0001-9407-1245)
- Philipp F. Lange (ORCID: https://orcid.org/0000-0003-1171-5864)
- Tess C. Lengyell (ORCID: https://orcid.org/0000-0001-6292-0724)
- Terri L. Petkau
- Pardis Kazemian (ORCID: https://orcid.org/0000-0001-8546-2471)
- Blair R. Leavitt (ORCID: https://orcid.org/0000-0002-4532-766X)
- Benjamin Life (ORCID: https://orcid.org/0000-0002-8221-3530)
- Elizabeth M. Simpson (ORCID: https://orcid.org/0000-0002-0654-4303)
- Alissandra de Moura Gomes
- Farah Jayousi
- Ning Shen
- Junia Myung
Institutions
- BC Children's Hospital (CA)
Publication Details
- Journal
- Neurobiology of Disease
- Published
- 2026-04-26
- DOI
- https://doi.org/10.1016/j.nbd.2026.107419
- Primary Topic
- Amyotrophic Lateral Sclerosis Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Weston Brain Institute
- Canadian Institutes of Health Research