Blood-Brain Barrier Penetrating Antisense Therapy For Spinal Muscular Atrophy

血脑屏障穿透反义疗法治疗脊髓性肌萎缩症

• 批准号: nhmrc : GNT1138033
• 负责人: A/Pr Bradley Turner
• 金额: $ 63.5万
• 依托单位国家: 澳大利亚
• 项目类别: Project Grants
• 财政年份: 2018
• 资助国家: 澳大利亚
• 起止时间: 2018-01-01 至 无数据
• 项目状态: 已结题
• 来源: https://researchdata.edu.au/blood-brain-barrier-muscular-atrophy/1348110
• 关键词: Blood; Brain; Barrier; Penetrating; Antisense

Spinal muscular atrophy (SMA) is a genetic disease caused by the deficiency of a protein known as survival motor neuron.This results in the degeneration of motor neurons (nerve cells controlling muscles) leading to progressive muscle weakness, paralysis, and eventual death. Currently, there is no known cure for SMA. The aim of proposed research is to develop gene-modifying molecules that prevent degeneration of motor neuron and extend the life-span of mice as a potential therapy for SMA.

脊髓性肌萎缩症（SMA）是一种遗传性疾病，由运动神经元存活蛋白缺乏引起，导致运动神经元（控制肌肉的神经细胞）退化，导致进行性肌肉无力、瘫痪，最终死亡。目前，SMA尚无已知的治愈方法。这项研究的目的是开发基因修饰分子，防止运动神经元变性，延长小鼠寿命，作为SMA的潜在治疗方法。