AAV9 gene therapy optimization for SMARD1/CMT2S: safety and long-term efficacy comparison of two vectors in a SMARD1 preclinical model
ICV gene therapy approach can effectively rescue SMARD1 pathological hallmarks, including astrogliosis and microgliosis. Moreover, P546-promoter construct is superior in terms of safety profile and long-term therapeutic efficacy.
Authors
- Giacomo P. Comi (ORCID: https://orcid.org/0000-0002-1383-5248)
- S. Corti
- Michela Taiana (ORCID: https://orcid.org/0000-0001-8257-8831)
- Linda Ottoboni (ORCID: https://orcid.org/0000-0001-5412-7785)
- Julieth A. Sierra-Delgado (ORCID: https://orcid.org/0000-0003-4316-6943)
- Manuela Garbellini (ORCID: https://orcid.org/0000-0003-0697-6381)
- Alessia Anastasia (ORCID: https://orcid.org/0000-0002-2404-4564)
- Elisa Pagliari (ORCID: https://orcid.org/0000-0002-9467-0595)
- Kathrin Meyer (ORCID: https://orcid.org/0000-0002-5052-2101)
- Jessica Ongaro (ORCID: https://orcid.org/0000-0003-2715-5355)
- Shibi Likhite
- Floriana Bellandi
- Monica Nizzardo
Institutions
- Nationwide Children's Hospital (US)
- University of Milan (IT)
- University of Missouri Health System (US)
- Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico (IT)
- Ospedale Maggiore (IT)
Publication Details
- Journal
- Journal of Biomedical Science
- Published
- 2026-01-04
- DOI
- https://doi.org/10.1186/s12929-025-01204-z
- Primary Topic
- Neurogenetic and Muscular Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Università degli Studi di Milano
- Dipartimenti di Eccellenza
- Fondazione Cariplo
- Ministero della Salute