Core--Animal

动物核心提供了一种中心机制，通过该机制提供了一致的、高质量的和成本有效的获得和开发小鼠遗传突变体的途径，用于与基因治疗和囊性纤维化相关的研究。这一核心支持灵长类动物的研究，提供在进行人体试验之前所需的过渡数据。它还有助于培训研究人员有效使用动物模型。该核心特别关注CFTR突变小鼠和突变小鼠的免疫/炎症反应的靶向改变;这些小鼠为与肺基因转移和肺慢性炎症和损伤相关的研究提供了重要模型，如CF中所见。还提供狒狒作为造血干细胞基因治疗的大型动物模型。

The animal core provides a central mechanism by which consistent, high quality and cost effective access to and development of mouse genetic mutants for studies related to gene therapy and cystic fibrosis are provided. This core supports studies in primates that provide transitional data needed before human trials can be undertaken. It also facilitates training of investigators in the efficient use of animal models. This core focuses, in particular, on provision of CFTR mutant mice and mutant mice with targeted alterations of their immune/inflammatory response; these mice provide important models for studies related to gene transfer to the lung and chronic inflammation and injury in the lung, as seen in CF. Baboons are also provided as a large animal model for hematopoietic stem cell gene therapy.