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Updated: Jun 18, 2025

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肝炎G2 PMM2-CDG淘汰模型:一种多功能平台,用于变种和治疗评估

Alicia Vilas1, Álvaro Briso-Montiano1, Cristina Segovia-Falquina1

  • 1Centro de Diagnóstico de Enfermedades Moleculares, Centro de Biología Molecular-SO UAM-CSIC, Universidad Autónoma de Madrid, Campus de Cantoblanco, 28049 Madrid, Spain; U746 - CIBER de Enfermedades Raras (CIBERER), Madrid, Spain; Instituto de Investigación Sanitaria IdiPAZ, Madrid, Spain.

Molecular genetics and metabolism
|August 3, 2024
PubMed
概括

通过CRISPR-Cas9基因编辑创建了基因突变酶2缺乏症 (PMM2-CDG) 的新细胞模型. 这种PMM2-CDG模型准确地复制了疾病特征,有助于变异评估和治疗策略开发.

关键词:
这就是CRISPR-Cas9的特征.疾病模型的疾病模型.这是一种HepG2型肝炎.这是一次 nokaut 的比赛.胺转基因酶2缺乏症 (PMM2-CDG) 的情况治疗策略 治疗策略变体评估变体评估

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