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A pilot project to demonstrate feasibility of a Canadian gene therapy clinical trials platform for rare genetic diseases: Gene Therapy for CD3delta Severe Combined Immune Deficiency.

A pilot project to demonstrate feasibility of a Canadian gene therapy clinical trials platform for rare genetic diseases: Gene Therapy for CD3delta Severe Combined Immune Deficiency.
一个试点项目，旨在证明加拿大罕见遗传病基因治疗临床试验平台的可行性：CD3delta 严重联合免疫缺陷的基因治疗。
批准号：
466428
负责人：
Wright Nicola A
金额：
$176.76万
依托单位：
University of Calgary
依托单位国家：
加拿大
项目类别：
Operating Grants
财政年份：
2022
资助国家：
加拿大
项目状态：
未结题
起止时间：
2022-07-01 至 2025-07-01
来源：
https://webapps.cihr-irsc.gc.ca/decisions/p/project_details.html?applId=466428&lang=en
关键词：
Adenine Base Editing Gene Editing Gene Therapy Clinical Trial Inborn Errors Of Immunity Rare Diseases Severe Combined Immune Deficiency

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中文摘要
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英文摘要
Gene therapies are a novel cure for rare genetic disorders. Currently, Canadian patients with inherited immune disorders eligible for gene therapies must travel to other countries for treatment, where cost can be over $2 million/patient. There is a signif
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A pilot project to demonstrate feasibility of a Canadian gene therapy clinical trials platform for rare genetic diseases: Gene Therapy for CD3delta Severe Combined Immune Deficiency.
  • 批准号：
    474614
  • 项目类别：
    Operating Grants
  • 资助金额：
    $44.19万
  • 财政年份：
    2022
  • 负责人：
    Wright Nicola A
  • 依托单位：
    University of Calgary
海外基金