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Updated: Jan 21, 2026

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一个工程UGA抑制器tRNA基因用于疾病无知的AAV传递

Mengyao Xu1, Hao Liu1, Jiaming Wang1

  • 1Department of Genetic and Cellular Medicine, University of Massachusetts Chan Medical School, Worcester, MA, USA.

Nature biotechnology
|January 19, 2026
PubMed
概括

通过腺相关病毒 (AAV) 传递的工程UGA抑制器转移RNAs (sup-tRNAs) 可以恢复基因疾病的小鼠模型中的酶功能. 这种方法为由特定突变引起的罕见疾病提供了一种新的治疗策略.

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