Gene Therapy as Treatment for Purine Nucleoside Phosphorylase Primary Immune Deficiency

基因疗法治疗嘌呤核苷磷酸化酶原发性免疫缺陷

• 批准号: 449530
• 负责人: Loves Robyn
• 金额: $ 1.27万
• 依托单位: University of Toronto
• 依托单位国家: 加拿大
• 项目类别: Studentship Programs
• 财政年份: 2020
• 资助国家: 加拿大
• 起止时间: 2020-12-01 至 2021-12-01
• 项目状态: 已结题
• 来源: https://webapps.cihr-irsc.gc.ca/decisions/p/project_details.html?applId=449530&lang=en
• 关键词: Gene Therapy; Hematology; Immunology; Metabolic Disorder; Pediatrics; Primary Immune Deficiency; Rare Diseases; Stem Cell Transplant; Stem Cells; Viral Vector

Patients born with purine nucleoside phosphorylase (PNP) deficiencies suffer from recurrent infections, autoimmunity, and cognitive and developmental delays since infancy. Many of the symptoms and complications that arise from the weakened immune system i

先天性嘌呤核苷磷酸化酶（PNP）缺陷的患者自婴儿期起就患有复发性感染、自身免疫以及认知和发育迟缓。许多症状和并发症是由免疫系统减弱引起的，