The Jackson Laboratory Center for Precision Genetics: From New Models to Novel Therapeutics

 描述（由申请人提供）：杰克逊实验室（JAX）精密遗传学中心（JCPG）将开发和传播新的，精确的动物模型的不治之症和遗传复杂的人类疾病。JCPG将利用JAX在哺乳动物遗传学和疾病建模方面的长期专业知识，以及由其新成立的临床基因组学研究所杰克逊基因组医学实验室（JAX-GM）培养的人类临床样本，数据和合作。JCPG的国际多学科团队-包括遗传学家和遗传学技术专家，分子和计算生物学家，特定疾病领域的临床专家以及精确疾病动物模型开发的世界领导者-将拥有集体能力，预测疾病建模需求，并将在区域，国家和国际基础上为各种疾病领域的研究人员提供服务。最初的六个项目将解决与遗传复杂性、分子遗传数据和/或代表临床前开发不同阶段的“人源化”相关的难治性或不治之症。联合政治协商小组将：目标1）将研究者发起的疾病模型研究项目与机构范围内的支持相结合，以创建高效和目标导向的临床前管道;目标2）在试点中心研究项目和核心中使用启发式程序开发，将JAX定位为五年内功能齐全的中心，同时在试点期间取得切实成果;目标3）根据完成工作的需要，既要拥抱现实的尖端技术平台，也要拥抱雄心勃勃的高风险、高回报平台，即，目标4）通过JAX的许多机构间合作，确定并吸引关键的临床、基金会和企业合作伙伴，他们与我们分享特定疾病目标的愿景，帮助我们实现更大的中心目标。最终，JCPG将产生新的疾病建模流程和管道、数据资源、研究结果和模型，这些将通过JAX经过验证的传播管道快速共享，以加速转化为医疗效益。

 DESCRIPTION (provided by applicant): The Jackson Laboratory (JAX) Center for Precision Genetics (JCPG) will develop and disseminate new, precise animal models of incurable and genetically complex human diseases. The JCPG will leverage JAX's long-established expertise in mammalian genetics and disease modeling as well as the human clinical samples, data and collaborations fostered by The Jackson Laboratory for Genomic Medicine (JAX-GM), its newly established clinical genomics institute. The JCPG's international, multi-disciplinary team-including geneticists and genetics technology experts, molecular and computational biologists, clinical experts in specific disease areas and world leaders in the development of precision animal models of disease-will possess the collective ability to foresee disease modeling needs as they emerge and will serve investigators in a variety of disease areas on a regional, national and international basis. Initially six projects will address intractable or incurable diseases linkd by genetic complexity, molecular genetic data and/or "humanization" that represent diverse stages of preclinical development. The JCPG will: Aim 1) Align investigator-initiated disease model research projects with institution-wide support to create efficient and goal-oriented preclinical pipelines; Aim 2) Use heuristic program development in the pilot center research projects and cores to position JAX to be a fully functional center in five years, while nevertheles achieving tangible outcomes during the pilot period; Aim 3) Embrace both realistic, cutting-edge technological platforms and ambitious high-risk, high-reward platforms as required to get the job done, i.e., advance paths to therapies for heretofore incurable diseases; and Aim 4) Through JAX's many inter-institutional collaborations, identify and engage key clinical, foundation and corporate partners, who share our vision both for specific disease objectives, to help us navigate larger center goals. Ultimately, the JCPG will generate new disease modeling processes and pipelines, data resources, research results and models that will be swiftly shared through JAX's proven dissemination pipelines to accelerate translation to medical benefit.