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对用于AAV基因疗法治疗单一性疾病的免疫抑制方案的系统审查

Besarte Vrellaku1, Ilda Sethw Hassan2, Rebecca Howitt3

  • 1Department of Paediatrics, MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre, University of Oxford, Oxford, UK.

Molecular therapy : the journal of the American Society of Gene Therapy
|July 24, 2024
PubMed
概括

基因相关病毒 (AAV) 基因疗法对遗传疾病具有前景,但免疫反应需要有效的免疫抑制. 本综述分析了改善AAV基因治疗安全性和有效性的协议.

关键词:
腺相关病毒,AAV.临床试验是指临床试验中的临床试验.基因治疗的基因疗法免疫力是一种免疫力.免疫抑制剂 免疫抑制剂免疫抑制协议 免疫抑制协议单源性疾病 单源性疾病

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Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...
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