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目前的AAV介导基因疗法的临床应用

Barry J Byrne¹, Kevin M Flanigan², Susan E Matesanz³

¹Powell Gene Therapy Center, University of Florida College of Medicine, Gainesville, FL, USA; Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL, USA.

Molecular therapy : the journal of the American Society of Gene Therapy

|May 7, 2025

概括

腺相关病毒 (AAV) 载体为遗传疾病提供了一个有前途的基因治疗方法,直接传递功能基因. 这次审查突出了AAV的重点.

科学领域:

生物技术是生物技术.
遗传学遗传学是一个
医学医学医学医学医学

背景情况:

遗传性疾病影响10%的人口,大多数治疗方法只能控制症状,而不是解决根本原因.
目前的基因疗法通常需要终身治疗,并且无法纠正潜在的遗传缺陷.
腺相关病毒 (AAV) 载体已经成为体内基因传递的领先技术.

研究的目的:

审查腺相关病毒 (AAV) 基因疗法的临床进展.
讨论AAV载体在治疗各种遗传疾病中的应用.
确定基于AAV的基因疗法的挑战和未来方向.

主要方法:

对当前临床AAV基因治疗文献的综述.
对在体内DNA输送的AAV载体效率的分析.
检查FDA批准的AAV产品和正在进行的临床试验.

主要成果:

AAV 载体是最有效的 in vivo DNA 输送到核的方法.
七种基于AAV的疗法已获得FDA批准,还有10多种在临床开发中.
AAV基因疗法正在应用于越来越多的遗传疾病.

结论:

AAV载体代表了遗传疾病治疗的重大进步.
临床应用范围包括神经肌肉,心血管,中枢神经系统,眼睛和代谢疾病.
克服现有的挑战将使AAV基因疗法的更广泛的临床采用成为可能.

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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...