Salk Institute for Biological Studies, 10010 N. Torrey Pines Rd., La Jolla, CA, 92037, USA.
Universidad Católica San Antonio de Murcia, Campus de los Jerónimos 135, Guadalupe, 30107, Spain.
Sci Rep. 2017 Sep 5;7(1):10487. doi: 10.1038/s41598-017-08596-5.
Genome editing using programmable nucleases has revolutionized biomedical research. CRISPR-Cas9 mediated zygote genome editing enables high efficient production of knockout animals suitable for studying development and relevant human diseases. Here we report efficient disabling pancreatogenesis in pig embryos via zygotic co-delivery of Cas9 mRNA and dual sgRNAs targeting the PDX1 gene, which when combined with chimeric-competent human pluriopotent stem cells may serve as a suitable platform for the xeno-generation of human tissues and organs in pigs.
利用可编程核酸酶进行基因组编辑已经彻底改变了生物医学研究。CRISPR-Cas9 介导的受精卵基因组编辑可高效产生适合研究发育和相关人类疾病的基因敲除动物。在此，我们报告了通过 Cas9 mRNA 和靶向 PDX1 基因的双 sgRNA 共递送来有效抑制猪胚胎胰腺发生的方法，当与嵌合能力的人类多能干细胞结合时，该方法可能成为在猪中异种生成人类组织和器官的合适平台。
