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Updated: Feb 11, 2026

Subpial Adeno-associated Virus 9 AAV9 Vector Delivery in Adult Mice
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在II型GM1liosidosis中AAV9基因治疗 - 一个阶段1-2试验

Connor J Lewis1,2, Precilla D'Souza1,2, Jean M Johnston1,2

  • 1Office of the Clinical Director, National Human Genome Research Institute, Bethesda, MD.

The New England journal of medicine
|February 10, 2026
PubMed
概括

使用AAV9治疗GM1liosidosis的基因治疗显示出潜力,改善了生物标志物和神经成像,但也出现了吐和肝酶升高等不良事件. 对这种致命的神经退行性疾病需要进一步的研究.

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