Nakamura Shingo, Inada Emi, Saitoh Issei, Sato Masahiro
Division of Biomedical Engineering, National Defense Medical College Research Institute, Saitama 359-8513, Japan.
Department of Pediatric Dentistry, Graduate School of Medical and Dental Sciences, Kagoshima University, Kagoshima 890-8544, Japan.
BioTech (Basel). 2023 May 11;12(2):37. doi: 10.3390/biotech12020037.
Genome editing, as exemplified by the CRISPR/Cas9 system, has recently been employed to effectively generate genetically modified animals and cells for the purpose of gene function analysis and disease model creation. There are at least four ways to induce genome editing in individuals: the first is to perform genome editing at the early preimplantation stage, such as fertilized eggs (zygotes), for the creation of whole genetically modified animals; the second is at post-implanted stages, as exemplified by the mid-gestational stages (E9 to E15), for targeting specific cell populations through in utero injection of viral vectors carrying genome-editing components or that of nonviral vectors carrying genome-editing components and subsequent in utero electroporation; the third is at the mid-gestational stages, as exemplified by tail-vein injection of genome-editing components into the pregnant females through which the genome-editing components can be transmitted to fetal cells via a placenta-blood barrier; and the last is at the newborn or adult stage, as exemplified by facial or tail-vein injection of genome-editing components. Here, we focus on the second and third approaches and will review the latest techniques for various methods concerning gene editing in developing fetuses.
以CRISPR/Cas9系统为例的基因组编辑技术，近来已被用于有效地生成转基因动物和细胞，以进行基因功能分析和创建疾病模型。在个体中诱导基因组编辑至少有四种方法：第一种是在植入前的早期阶段，如受精卵（合子）阶段进行基因组编辑，以创建完整的转基因动物；第二种是在植入后的阶段，如妊娠中期（E9至E15），通过子宫内注射携带基因组编辑组件的病毒载体或携带基因组编辑组件的非病毒载体并随后进行子宫内电穿孔，来靶向特定细胞群体；第三种是在妊娠中期，如通过尾静脉向怀孕雌性动物注射基因组编辑组件，基因组编辑组件可通过胎盘-血液屏障传递给胎儿细胞；最后一种是在新生或成年阶段，如通过面部或尾静脉注射基因组编辑组件。在此，我们重点关注第二种和第三种方法，并将综述有关发育中胎儿基因编辑的各种方法的最新技术。
