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Huifang Peng

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#gene editing Review Open access Aug 2026

Gene and nucleic acid therapies for MMUT-deficient methylmalonic acidemia: from AAV-mediated gene addition to LNP-mediated mRNA delivery and precision genome editing

Current therapeutic evidence is concentrated primarily in MMUT- deficient isolated MMA, and further progress will require safer and more durable delivery platforms, improved tissue targeting, robust long-term safety assessment, clinically meaningful endpoints, and careful evaluation of accessibility and cost.

Yan-Qiu Wang, Huifang Peng, T. Jiang et al. · 0 citations

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