IN UTERO SMALL AND LARGE ANIMAL RESOURCE CORE

项目总结 许多单基因疾病在出生前或出生后不久发作，并导致显著的发病率和死亡率。在子宫内 基因组编辑(IUGE)利用了正常的胎儿属性，包括体积小，祖细胞丰富 细胞和未成熟的免疫系统，以安全和有效地在 病理学的开始。为了充分了解IUGE的潜力并为IND应用做准备，严格 需要对小动物和大动物进行研究，以评估疗效以及胎儿和产妇的安全性。最新消息 子宫大小动物资源核心将提供所需的知识和技术专长 在小鼠和大型动物胎儿模型中设计、实施和分析体内基因编辑疗法，包括 非人灵长类动物。

PROJECT SUMMARY Many monogenic diseases strike prior to or soon after birth and cause significant morbidity and mortality. In utero genome editing (IUGE) takes advantage of normal fetal properties, including small size, abundant progenitor cells, and immature immune system, to safely and efficiently institute the genome editing therapeutic prior to the onset of pathology. To fully understand the potential of IUGE and in preparation for IND applications, rigorous small and large animal studies are required to assess efficacy as well as fetal and maternal safety. The In Utero Small and Large Animal Resource Core will provide the knowledge and technical expertise required to design, implement and analyze in vivo gene editing therapies in mouse and large animal fetal models, including nonhuman primates.