利用 Cas9 在成年小鼠中进行基因组编辑可纠正疾病突变和表型。
Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype.
机构信息
1] David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, Massachusetts, USA. [2].
David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, Massachusetts, USA.
出版信息
Nat Biotechnol. 2014 Jun;32(6):551-3. doi: 10.1038/nbt.2884. Epub 2014 Mar 30.
Abstract
We demonstrate CRISPR-Cas9-mediated correction of a Fah mutation in hepatocytes in a mouse model of the human disease hereditary tyrosinemia. Delivery of components of the CRISPR-Cas9 system by hydrodynamic injection resulted in initial expression of the wild-type Fah protein in ∼1/250 liver cells. Expansion of Fah-positive hepatocytes rescued the body weight loss phenotype. Our study indicates that CRISPR-Cas9-mediated genome editing is possible in adult animals and has potential for correction of human genetic diseases.
摘要
我们展示了 CRISPR-Cas9 介导的在遗传性酪氨酸血症的小鼠模型中肝细胞中 Fah 突变的纠正。通过水力注射递送 CRISPR-Cas9 系统的组件导致野生型 Fah 蛋白在约 1/250 的肝细胞中最初表达。Fah 阳性肝细胞的扩增挽救了体重减轻表型。我们的研究表明，CRISPR-Cas9 介导的基因组编辑在成年动物中是可行的，并具有纠正人类遗传疾病的潜力。
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