AAV基因治疗粘多糖病。

IF 12.8 Q1 MEDICINE, RESEARCH & EXPERIMENTAL

Med Pub Date : 2025-04-11 DOI:10.1016/j.medj.2025.100609

Shaukat Khan, Yasuhiko Ago, Shunji Tomatsu

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引用次数: 0

摘要

腺相关病毒（AAV）载体是一种很有前途的遗传病治疗选择。由于其安全性、有效性和提供长期治疗性转基因表达的能力，它们被认为是最有效的体内基因疗法。1,2,3 Rossi等人1已经证明AAV基因治疗粘多糖病是有效和安全的，为粘多糖病的潜在治疗铺平了道路。

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AAV gene therapy for mucopolysaccharidoses.

Adeno-associated viral (AAV) vectors emerge as a promising treatment option for genetic diseases. They are recognized as the most effective in vivo gene therapy due to their safety, efficacy, and ability to provide long-term therapeutic transgene expression.¹^,²^,³ Rossi et al.¹ have demonstrated that AAV gene therapy is effective and safe for treating patients with mucopolysaccharidosis VI, paving the way for potential treatments for mucopolysaccharidoses.

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来源期刊

Med MEDICINE, RESEARCH & EXPERIMENTAL-

CiteScore

17.70

自引率

0.60%

发文量

102

期刊介绍： Med is a flagship medical journal published monthly by Cell Press, the global publisher of trusted and authoritative science journals including Cell, Cancer Cell, and Cell Reports Medicine. Our mission is to advance clinical research and practice by providing a communication forum for the publication of clinical trial results, innovative observations from longitudinal cohorts, and pioneering discoveries about disease mechanisms. The journal also encourages thought-leadership discussions among biomedical researchers, physicians, and other health scientists and stakeholders. Our goal is to improve health worldwide sustainably and ethically. Med publishes rigorously vetted original research and cutting-edge review and perspective articles on critical health issues globally and regionally. Our research section covers clinical case reports, first-in-human studies, large-scale clinical trials, population-based studies, as well as translational research work with the potential to change the course of medical research and improve clinical practice.