Wang Dan, Niu Yuyu, Ren Lingzhi, Kang Yu, Tai Phillip W L, Si Chenyang, Mendonca Craig A, Ma Hong, Gao Guangping, Ji Weizhi
Horae Gene Therapy Center University of Massachusetts Medical School Worcester MA 01605 USA.
Department of Microbiology and Physiological Systems University of Massachusetts Medical School Worcester MA 01605 USA.
Adv Sci (Weinh). 2019 Sep 4;6(21):1900440. doi: 10.1002/advs.201900440. eCollection 2019 Nov 6.
Delivery of genome editing tools to mammalian zygotes has revolutionized animal modeling. However, the mechanical delivery method to introduce genes and proteins to zygotes remains a challenge for some animal species that are important in biomedical research. Here, an approach to achieve gene delivery and genome editing in nonhuman primate embryos is presented by infecting zygotes with recombinant adeno-associated viruses (rAAVs). Together with previous reports from the authors of this paper and others, this approach is potentially applicable to a broad range of mammals. In addition to genome editing and animal modeling, this rAAV-based method can facilitate gene function studies in early-stage embryos.
将基因组编辑工具递送至哺乳动物受精卵,彻底改变了动物模型构建。然而,对于一些在生物医学研究中具有重要意义的动物物种而言,通过机械方式向受精卵中导入基因和蛋白质的方法仍然是一项挑战。在此,本文介绍了一种通过用重组腺相关病毒(rAAV)感染受精卵,从而在非人灵长类胚胎中实现基因递送和基因组编辑的方法。结合本文作者及其他作者之前的报道,该方法可能适用于广泛的哺乳动物。除了基因组编辑和动物模型构建外,这种基于rAAV的方法还可促进早期胚胎中的基因功能研究。