Core--Animal

动物核心提供了一种核心机制，通过该机制，为与基因治疗和囊性纤维化相关的研究提供了一致、高质量和成本效益的获得和开发小鼠基因突变的途径。这一核心支持对灵长类动物的研究，这些研究提供了在进行人体试验之前所需的过渡数据。它还有助于对研究人员进行有效使用动物模型的培训。这一核心尤其侧重于为CFTR突变小鼠和突变小鼠提供其免疫/炎症反应的有针对性的改变；这些小鼠为与肺内基因转移和肺内慢性炎症和损伤相关的研究提供了重要的模型，如在CF中所见。此外，还提供了一种用于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.