Development of a Gene Therapy with Lentiviral Vectors for Spinocerebellar Ataxias
Tiago Moreira-Gomes1,2,3, Clévio Nóbrega4,5
1ABC-Ri, Algarve Biomedical Center Research Institute, Faro, Portugal.
Methods in Molecular Biology (Clifton, N.J.)
|November 22, 2025
Abstract:
Among viral vectors, lentiviral vectors allow an efficient delivery of nucleic acid to the target cells due to its ability of gene integration into host genome and broad tropism. In this sense, this tool demonstrated to be very promising in genetic disorders, such as polyglutamine spinocerebellar ataxias. Therefore, we describe a protocol that explains in detail the production, purification, and injection of lentiviral vectors in a spinocerebellar ataxia mouse model.
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