Preclinical validation of AAV9-TECPR2 gene therapy in a novel knock-in model of TECPR2-related disorder
TECPR2 deficiency disrupts brainstem sensory-motor circuits, impairing autophagy and tactile, gait, and startle function and is prevented by neonatal AAV9.
Authors
- Bruna Lenfers Turnes (ORCID: https://orcid.org/0000-0002-3989-8394)
- Leo Mejia
- A S Kopin
- Nick Andrews (ORCID: https://orcid.org/0000-0003-2069-2684)
- Nathaniel Hodgson (ORCID: https://orcid.org/0000-0001-8299-3963)
- Darred Surin
- Francesco Villa (ORCID: https://orcid.org/0000-0002-1696-6657)
- Michela Fagiolini
- Maryam Arab
- Emma Cropper
- Tiffany Berry
- Peter Casey-Caplan
- Silmara de Lima
- Darius Ebrahimi-Fakhari
- Biyao Zhang
- Julie Zhao
Institutions
- Boston University (US)
- Tufts University (US)
- Boston Children's Hospital (US)
- Salk Institute for Biological Studies (US)
- Harvard University (US)
- Boston Children's Museum (US)
- Pittsburg State University (US)
Publication Details
- Journal
- bioRxiv (Cold Spring Harbor Laboratory)
- Published
- 2026-03-04
- DOI
- https://doi.org/10.64898/2026.03.02.708636
- Primary Topic
- Hereditary Neurological Disorders
- Type
- article
- Field-Weighted Citation Impact
- 0.00