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Updated: Jul 15, 2025

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治疗多神经病的遗传性转基因粉症

Teresa Coelho1, Wilson Marques2, Noel R Dasgupta3

  • 1Centro Hospitalar Universitário de Santo António, Porto, Portugal.

JAMA
|September 28, 2023
PubMed
概括

艾普隆特森显著降低了血清中氨酸度,改善了遗传性氨酸氨酸多性神经病患者的神经病变和生活质量. 这种基因沉默疗法提供了一个有前途的新疗法.

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