Evaluation of cAbl allosteric inhibitors in Gaucher-Parkinson

戈谢-帕金森病 cAbl 变构抑制剂的评价

• 批准号: 10916051
• 负责人: Mark Henderson
• 金额: $ 12.58万
• 依托单位: NATIONAL CENTER FOR ADVANCING TRANSLATIONAL SCIENCES
• 依托单位国家: 美国
• 资助国家: 美国
• 起止时间: 至
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10916051
• 关键词: Animal Model; Automobile Driving; Basic Science; Collaborations; Disease; Evaluation; Goals; Health; Human; Human Genome; National Center for Advancing Translational Sciences; Nerve Degeneration; Parkinson Disease; Phosphotransferases; Rare Diseases; Research; Role; Translations; alpha synuclein; drug candidate; efficacy study; improved; inhibitor; mouse model; mutant; neurogenesis; pharmacologic; programs; tool

The main goal of this collaboration is to understand the role in cAbl kinase in the neurodegeneration associated with GBA1 mutants in Parkinson disease and evaluate cAbl allosteric modulators as potential treatment for this disease using mouse models. During this period, the collaborative team developed a research plan to interrogate the role of cAbl in neurogenesis, including animal models of Parkinsons. Initial efficacy studies in murine models of A53T SNCA and GBA1-associated Parkinson disease were completed, and promising activity was observed.

这项合作的主要目标是了解cAbl激酶在帕金森病中与GBA 1突变体相关的神经变性中的作用，并使用小鼠模型评估cAbl变构调节剂作为这种疾病的潜在治疗方法。 在此期间，合作团队制定了一项研究计划，以询问cAbl在神经发生中的作用，包括帕金森病的动物模型。在A53T SNCA和GBA 1相关帕金森病小鼠模型中完成了初步疗效研究，并观察到了有希望的活性。