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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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Development of a Gene Therapy with Lentiviral Vectors for Spinocerebellar Ataxias

  • 实验方案

  • 发表日期：

    2026年

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  • Tiago Moreira-Gomes,
  • Clévio Nóbrega

摘要

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.