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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    Studysummary This study found that minicircle-based gene therapy significantly lowered total homocysteine levels and improved liver CBS activity in a mouse model of CBS deficiency.
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