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Evaluation of cAbl allosteric inhibitors in Gaucher-Parkinson

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
项目类别：
财政年份：
--
资助国家：
美国
项目状态：
未结题
起止时间：
至
来源：
https://reporter.nih.gov/project-details/10916051
关键词：
Animal ModelAutomobile DrivingBasic ScienceCollaborationsDiseaseEvaluationGoalsHealthHumanHuman GenomeNational Center for Advancing Translational SciencesNerve DegenerationParkinson DiseasePhosphotransferasesRare DiseasesResearchRoleTranslationsalpha synucleindrug candidateefficacy studyimprovedinhibitormouse modelmutantneurogenesispharmacologicprogramstool

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中文摘要
翻译
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。
英文摘要
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.
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