推进亨廷顿病的 RNA 疗法-猫眼课题宝

推进亨廷顿病的 RNA 疗法

批准号：

10359054

负责人：

NEIL ARONIN

金额：

$125.61万

依托单位：

UNIV OF MASSACHUSETTS MED SCH WORCESTER

依托单位国家：

美国

项目类别：

财政年份：

2020

资助国家：

美国

项目状态：

未结题

起止时间：

2020-02-01 至 2025-01-31

来源：

https://reporter.nih.gov/project-details/10359054

关键词：

Affect Age Amyotrophic Lateral Sclerosis Animal Disease Models Biological Assay Blood Chemical Analysis Bolus Infusion Brain Brain Diseases Brain region CAG repeat Care given by nurses Cerebral Dominance Cerebral Ventricles Cerebrospinal Fluid Chemicals Chemistry Child Complete Blood Count Coupling Crowding DARPP Data Dementia Discipline of Nursing Disease Dose Drug Stability Dyskinetic syndrome Economic Burden Economics Evaluation Exhibits Family Female Filtration Formulation Frontotemporal Dementia Genes Genetic Glial Fibrillary Acidic Protein Goals Gross National Product Head Health Benefit High Pressure Liquid Chromatography Human Huntington Disease Huntington gene Huntington protein Hydrophobicity Impaired cognition Individual Inherited Injections Lateral Magnetic Resonance Imaging Measures Mental Depression Messenger RNA Modification Movement Mus Mutation Neostriatum Neurodegenerative Disorders Neurons Onset of illness Outcome Outcome Measure Parents Patients Process Production Property Proteins Public Health RNA Interference Route Safety Secure Severity of illness Small Interfering RNA Societies Spinal Stress Structure Testing Therapeutic Time Toxic effect Transgenic Organisms Western Blotting Work base cytokine drug testing effective therapy flexibility in vivo lateral ventricle lead candidate male motor impairment mutant nervous system disorder neuropathology nonhuman primate phosphoramidite polyglutamine pressure process optimization programs public health relevance research clinical testing response therapeutic RNA uptake

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ABSTRACT The cause of Huntington’s disease is an increase in the trinucleotide CAG repeat from under 36 repeats to 36 or greater repeats. The mode for the number of repeats is 42, and most patients have between 40 and 45. The disease generally starts between ages 30 and 40, with onset and progression of impaired cognition, depression, and aberrant movement. The genetics is autosomal dominant. Lowering mutant huntingtin in HD animal models delays onset of disease or mitigates the severity of disease. We use advanced, modified, di- branched siRNA to lower mutant huntingtin, by invoking RNA interference in brain. Our advanced siRNA achieves therapeutic advantages: spread throughout the brain in non- human primate and long-term huntingtin lowering after a single administration into the cerebrospinal fluid. The siRNA will be optimized in structure for safety and preliminary results will be secured. The goal of this CREATE proposal is to set the stage for promising therapeutics for treatment of Huntington’s disease. Similar treatments could be applicable to other autosomal dominant neurological disorders.

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