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H. Jiang

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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
Open access Aug 2026

Reclassification of the GRIA3 splice-site variant in an X-linked family with intellectual disability and psychiatric symptoms

This variant is the first reported, enriching the database and providing additional evidence to support genetic counselling and prenatal diagnosis, and is the first reported to lead to exon 2 skipping of GRIA3.

Lina Hu, Yuqiong Chai, Xiaofei Liu et al. · 0 citations