Treatment of Cystathionine Beta-Synthase Deficiency in Mice Using a Minicircle-Based Naked DNA Vector

    May 2019 in “ Human gene therapy ”
    Hyung‐Ok Lee, Lorena Gallego, Hiu Man Grisch‐Chan, Johannes Häberle, Beat Thöny, Warren D. Kruger
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    TLDR MC-DNA vector-based gene therapy can temporarily treat CBS deficiency in mice.
    The study demonstrated that a minicircle-based naked DNA vector (MC.P3-hCBS) could effectively treat cystathionine β-synthase (CBS) deficiency in mice. Researchers injected 24 Tg-1278T Cbs mice, with 15 receiving 50 µg of MC.P3-hCBS, resulting in a significant reduction in serum total homocysteine levels by 64% at day 21 post-injection and increased liver CBS activity. The treatment also ameliorated the alopecia phenotype, with treated mice showing thicker hair and whiskers. However, the effects were transient, with homocysteine levels returning to near pre-injection levels by day 98. The study concluded that MC-DNA vector-based gene therapy could be a potential treatment for CBS deficiency, despite the temporary nature of the effects and the stress associated with the injection method.
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