Reduction of ATXN2 expression as a means to treat Spinocerebellar Ataxia Type 2 (SCA2)

减少 ATXN2 表达作为治疗 2 型脊髓小脑共济失调 (SCA2) 的方法

• 批准号: 9359870
• 负责人: Ajit Jadhav
• 金额: $ 12.2万
• 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/9359870
• 关键词: Biology; Chemicals; Collaborations; Communities; Development; Disease; Extramural Activities; Fostering; General Population; Genes; Genomics; Goals; Informatics; Lead; Modeling; Mutation; Neurodegenerative Disorders; Pharmaceutical Chemistry; Pharmaceutical Preparations; Process; Publications; Research; Research Personnel; Resources; Synthesis Chemistry; Therapeutic; Type 2 Spinocerebellar Ataxia; United States National Institutes of Health; Validation; Work; assay development; base; drug development; drug discovery; high throughput screening; human disease; improved; inhibitor/antagonist; mutant; new technology; novel therapeutics; screening; small molecule; small molecule therapeutics; therapeutic development; tool

During this period, the NCGC conducted the primary screen and identified putative inhibitors of ATXN2 expression, which are currently being validated. As a center, the NCGC has fostered and maintained over 110 active collaborations with both NIH and extramural investigators, facilitating drug discovery efforts across the entire spectrum of human disease. These efforts have led to dozens of high-throughput screens and a number of medicinal chemistry campaigns to further improve on screening hits, providing our collaborators and the general research community with publications and a variety of promising small molecule probes and leads. In addition, the NCGC has worked to advance a number of informatic initiatives to make better use of existing drug and disease target information and provide the general public with easily accessible resources, further catalyzing the development of new therapies for human disease.

在此期间，NCGC进行了初步筛选，并确定了ATXN2表达的推定抑制剂，目前正在验证中。 作为一个中心，NCGC与NIH和校外研究人员建立并保持了110多个积极的合作关系，促进了整个人类疾病领域的药物发现工作。 这些努力已经促成了数十项高通量筛选和许多药物化学活动，以进一步改进筛选结果，为我们的合作者和一般研究界提供出版物和各种有前途的小分子探针和线索。 此外，NCGC还致力于推进多项信息化举措，以更好地利用现有的药物和疾病靶点信息，并为公众提供易于获取的资源，进一步促进人类疾病新疗法的开发。