RNAi therapy for Huntingtons disease: safety & efficacy in the nonhuman primate

亨廷顿病的 RNAi 疗法：安全性

• 批准号: 8270977
• 负责人: Jodi L. McBride
• 金额: $ 24.9万
• 依托单位: OREGON HEALTH & SCIENCE UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2011
• 资助国家: 美国
• 起止时间: 2011-06-15 至 2014-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8270977
• 关键词: 18q; Adult; Affect; Aggressive behavior; Alleles; Animals; Anxiety; Area; Behavioral; Behavioral Assay; Binding; Biological Assay; Brain; Brain region; CAG repeat; Chorea; Clinic; Cognition; Cognitive; Cognitive deficits; Corpus striatum structure; Dancing; Data; Development; Disease; Emotional; Emotional Disturbance; Emotions; Evaluation; Exons; Functional disorder; Genes; Genetic; Genetic Polymorphism; Glutamine; Human; Huntington Disease; In Vitro; Injection of therapeutic agent; Knockout Mice; Left; Longevity; Macaca mulatta; Magnetic Resonance Imaging; Mediating; Mental Depression; Messenger RNA; Methods; MicroRNAs; Modeling; Molecular; Monkeys; Motor; Mus; Mutate; Neck; Nerve Degeneration; Neurodegenerative Disorders; Nucleotides; Pathology; Patients; Play; Postdoctoral Fellow; Prevention; RNA Interference; Recombinant adeno-associated virus (rAAV); Research Personnel; Rodent Model; Role; Safety; Short-Term Memory; Symptoms; Testing; Therapeutic; Therapeutic Effect; Toxic effect; Training; Transgenic Mice; United States; Viral; Virus; abstracting; caudate nucleus; design; experience; gain of function; human Huntingtin protein; in vivo; limb movement; motor control; motor deficit; mutant; neuropathology; nonhuman primate; novel; pre-clinical; prevent; programs; psychologic; putamen; recombinant viral vector; response