Core--Animal

核心--动物

• 批准号: 7337075
• 负责人: Christopher B. Wilson
• 金额: $ 9.23万
• 依托单位: UNIVERSITY OF WASHINGTON
• 依托单位国家: 美国
• 财政年份: 2007
• 资助国家: 美国
• 起止时间: 2007-01-01 至 2008-12-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7337075
• 关键词: Animal Model; Animals; Area; Bone Marrow; Chronic; Clinical Data; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Data; Development; Gene Transfer; Generations; Genetic; Genotype; Hematopoietic stem cells; Human; Immune; Immune response; Individual; Infection; Inflammation; Inflammatory Response; Injury; Knockout Mice; Lung; Modeling; Mus; Mutant Strains Mice; Papio; Preparation; Primates; Production; Proteins; Risk; Study models; Transgenic Mice; cost effective; gene therapy; in vivo; injured; investigator training; mouse model; mutant; pre-clinical; response; tool; vector

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.

动物核心提供了一种中心机制，通过该机制可以一致、高质量且具有成本效益地获取和开发小鼠基因突变体，以用于基因治疗和囊性纤维化相关的研究。该核心支持灵长类动物的研究，提供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。该核心尤其侧重于提供 CFTR 突变小鼠和有针对性地改变其免疫/炎症反应的突变小鼠；这些小鼠为与肺部基因转移以及肺部慢性炎症和损伤相关的研究提供了重要模型，如 CF 中所示。狒狒也被提供作为造血干细胞基因治疗的大型动物模型。