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使用腺病毒载体进行血友病的临床前基因治疗研究。

Preclinical gene therapy studies for hemophilia using adenoviral vectors.

作者信息

Thorrez Lieven, VandenDriessche Thierry, Collen Désiré, Chuah Marinee K

机构信息

Center for Transgene Technology and Gene Therapy, University of Leuven, Flanders Interuniversity Institute for Biotechnology (VIB), University Hospital Gasthuisberg, Leuven, Belgium.

出版信息

Semin Thromb Hemost. 2004 Apr;30(2):173-83. doi: 10.1055/s-2004-825631.

Abstract

Hemophilia A and B are hereditary coagulation defects resulting from a deficiency of factor VIII (FVIII) and factor IX (FIX), respectively. Introducing a functional FVIII or FIX gene could potentially provide a cure for these bleeding disorders. Adenoviral vectors have been used as tools to introduce potentially therapeutic genes into mammalian cells and are by far the most efficient vectors for hepatic gene delivery. Long-term expression of both FVIII and FIX has been achieved in preclinical (hemophilic) mouse models using adenoviral vectors. Therapeutic levels of FVIII and FIX also have been achieved in hemophilic dogs using adenoviral vectors and in some cases expression was long-term. The performance of earlier generation adenoviral vectors, which retained residual viral genes, was compromised by potent acute and chronic inflammatory responses that contributed to significant toxicity and morbidity and short-term expression of FVIII and FIX. The development of improved adenoviral vectors devoid of viral genes (gutless or high-capacity adenoviral vectors) was therefore warranted, which led to a significant reduction in acute and chronic toxicity and more prolonged expression of FVIII and FIX. Strategies aimed at making these vectors safer and less immunogenic and their implications for hemophilia gene therapy are discussed in this review.

摘要

甲型血友病和乙型血友病是分别由于缺乏凝血因子VIII(FVIII)和凝血因子IX(FIX)而导致的遗传性凝血缺陷。引入功能性FVIII或FIX基因可能为这些出血性疾病提供治愈方法。腺病毒载体已被用作将潜在治疗性基因导入哺乳动物细胞的工具,并且是迄今为止用于肝脏基因递送的最有效载体。使用腺病毒载体在临床前(血友病)小鼠模型中已实现FVIII和FIX的长期表达。在血友病犬中使用腺病毒载体也已达到FVIII和FIX的治疗水平,并且在某些情况下表达是长期的。保留残留病毒基因的早期一代腺病毒载体的性能受到强烈的急性和慢性炎症反应的影响,这些反应导致了显著的毒性和发病率以及FVIII和FIX的短期表达。因此,有必要开发不含病毒基因的改良腺病毒载体(无内脏或高容量腺病毒载体),这导致急性和慢性毒性显著降低以及FVIII和FIX的表达时间延长。本综述讨论了旨在使这些载体更安全、免疫原性更低的策略及其对血友病基因治疗的影响。

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