Single-dose CRISPR-Cas9 therapy extends lifespan of mice with Hutchinson-Gilford progeria syndrome
Single-dose CRISPR-Cas9 therapy extends lifespan of mice with Hutchinson-Gilford progeria syndrome
复制标题
DOI：
10.1038/s41591-019-0343-4
复制
发表时间：
2019-03-01
期刊：
影响因子：
82.9
通讯作者：
Belmonte, Juan Carlos Izpisua
中科院分区：
文献类型：
--
作者：
Beyret, Ergin;Liao, Hsin-Kai;Belmonte, Juan Carlos Izpisua
Hutchinson-Gilford早衰综合征（HGPS）是一种罕见的致命遗传性疾病，其特征是症状让人联想到加速衰老。主要的潜在遗传原因是核纤层蛋白A编码基因的替代突变，导致产生一种名为早老蛋白的有毒亚型。在这里，我们表明，通过单剂量全身施用腺相关病毒递送的CRISPR-Cas9组分来减少核纤层蛋白A/早老蛋白抑制了小鼠模型中的HGPS。
Hutchinson-Gilford progeria syndrome (HGPS) is a rare lethal genetic disorder characterized by symptoms reminiscent of accelerated aging. The major underlying genetic cause is a substitution mutation in the gene coding for lamin A, causing the production of a toxic isoform called progerin. Here we show that reduction of lamin A/progerin by a single-dose systemic administration of adeno-associated virus-delivered CRISPR-Cas9 components suppresses HGPS in a mouse model.