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Gene Therapy as Treatment for Purine Nucleoside Phosphorylase Primary Immune Deficiency

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
项目状态：
已结题
起止时间：
2020-12-01 至 2021-12-01
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

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中文摘要
翻译
先天性嘌呤核苷磷酸化酶（PNP）缺乏症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。免疫系统减弱引起的许多症状和并发症
英文摘要
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
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Gene therapy for pediatric primary immune deficiency
Gene Therapy as Treatment for Purine Nucleoside Phosphorylase Primary Immune Deficiency
  • 批准号：
    428969
  • 项目类别：
    Studentship Programs
  • 资助金额：
    $1.27万
  • 财政年份：
    2019
  • 负责人：
    Loves Robyn
  • 依托单位：
    University of Toronto
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