Innovative Genomics Institute, University of California, Berkeley, California 94720, United States; California Institute for Quantitative Biosciences, University of California, Berkeley, California 94720, United States; ORCID https://orcid.org/0000-0003-2937-4703.
Innovative Genomics Institute, University of California, Berkeley, California 94720, United States; California Institute for Quantitative Biosciences, University of California, Berkeley, California 94720, United States; ORCID https://orcid.org/0000-0002-0644-5540.
Trends Mol Med. 2018 Oct;24(10):822-824. doi: 10.1016/j.molmed.2018.07.008. Epub 2018 Aug 10.
Despite the unparalleled therapeutic promise of genome editing, its curative power is currently limited by the substantial difficulty in delivering DNA-cutting enzymes to the cells in need of correction. A recent study demonstrates the potential for the delivery of pre-assembled genome-editing enzymes in the form of ribonucleoprotein complexes, which were used to rescue a mouse model of fragile X syndrome (FXS).
尽管基因组编辑具有无与伦比的治疗潜力，但目前其治疗效果受到将 DNA 切割酶递送到需要校正的细胞中的实质性困难的限制。最近的一项研究表明，以核糖核蛋白复合物的形式递送电组装基因组编辑酶具有潜力，该复合物用于拯救脆性 X 综合征（FXS）的小鼠模型。
