基于 CRISPR/Cas9 的亨廷顿病戈谢病综合征治疗方法的开发。
Development of a CRISPR/Cas9-based therapy for Hutchinson-Gilford progeria syndrome.
机构信息
Departamento de Bioquímica y Biología Molecular, Facultad de Medicina, Instituto Universitario de Oncología del Principado de Asturias, Universidad de Oviedo, Oviedo, Spain.
Centro de Investigación Biomédica en Red de Cáncer, Madrid, Spain.
出版信息
Nat Med. 2019 Mar;25(3):423-426. doi: 10.1038/s41591-018-0338-6. Epub 2019 Feb 18.
CRISPR/Cas9-based therapies hold considerable promise for the treatment of genetic diseases. Among these, Hutchinson-Gilford progeria syndrome, caused by a point mutation in the LMNA gene, stands out as a potential candidate. Here, we explore the efficacy of a CRISPR/Cas9-based approach that reverts several alterations in Hutchinson-Gilford progeria syndrome cells and mice by introducing frameshift mutations in the LMNA gene.
基于 CRISPR/Cas9 的疗法为治疗遗传疾病带来了巨大的希望。在这些疾病中，由 LMNA 基因突变引起的亨廷顿氏舞蹈症是一个潜在的候选疾病。在这里，我们探讨了一种基于 CRISPR/Cas9 的方法的疗效，该方法通过在 LMNA 基因中引入移码突变，纠正亨廷顿氏舞蹈症细胞和小鼠中的几种改变。
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