Long-lasting recovery of lysosomal acid lipase deficiency using non-viral delivery of non-integrating episomal DNA constructs.

使用非整合附加型 DNA 构建体的非病毒递送来长期恢复溶酶体酸性脂肪酶缺乏症。

• 批准号: 478865
• 负责人: Brambilla Davide
• 金额: $ 48.21万
• 依托单位: Universit de Montral
• 依托单位国家: 加拿大
• 项目类别: Operating Grants
• 财政年份: 2023
• 资助国家: 加拿大
• 起止时间: 2023-03-01 至 2027-03-01
• 项目状态: 未结题
• 来源: https://webapps.cihr-irsc.gc.ca/decisions/p/project_details.html?applId=478865&lang=en
• 关键词: Drug Delivery; Gene Delivery; Lipid Metabolism; Nanoparticles; Rare Disease

Several diseases are the consequence of one or more mutations in the genetic material of the cells that constitute organs. In some cases, these mutations take place in DNA regions coding for proteins, such as enzymes, leading to the formation of altered p

有几种疾病是构成器官的细胞的遗传物质发生一种或多种突变的结果。在某些情况下，这些突变发生在编码蛋白质（如酶）的DNA区域，导致形成改变的蛋白质。