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Additional file 1 of AAV9 gene therapy optimization for SMARD1/CMT2S: safety and long-term efficacy comparison of two vectors in a SMARD1 preclinical model

Year: 2026

Type: article

Source: Figshare

DOI: 10.6084/m9.figshare.30997126

Authors: Pagliari, Elisa, Anastasia, Alessia, Bellandi, Floriana, Garbellini, Manuela, Ongaro, Jessica, Taiana, Michela, Comi, Giacomo P., Ottoboni, Linda, Sierra-Delgado, Julieth Andrea, Likhite, Shibi, Meyer, Kathrin C., Nizzardo, Monica, Corti, Stefania P.

Institutions: University of Milan, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Nationwide Children's Hospital

Language: English

Topic: Virus-based gene therapy research

Subfield: Genetics

Field: Biochemistry, Genetics and Molecular Biology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: green

OpenAlex ID: W7119153374

FWCI:

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