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器官灌注体外递送病毒载体用于心脏移植基因治疗。

Ex Vivo Delivery of Viral Vectors by Organ Perfusion for Cardiac Transplantation Gene Therapy.

机构信息

Duke University, Durham, NC, USA.

TransMedics, Inc, Andover, MA, USA.

出版信息

Methods Mol Biol. 2022;2573:249-259. doi: 10.1007/978-1-0716-2707-5_19.

Abstract

Recent advances in ex vivo perfusion have enabled an extended preservation time for solid organs prior to transplantation allowing for possible resuscitation of the donor organ during the preservation period. Opportunities to provide viral vector-mediated gene therapy to the entire cardiac graft during this extended preservation period may lead to improvements in cardiac transplantation outcomes. Here we describe how to achieve successful gene delivery using viral vectors to an entire cardiac graft by normothermic, ex vivo perfusion. This protocol has been confirmed with the most highly utilized viral vector types in gene therapy clinical studies (adenoviral [Ad] and adeno-associated viral vector [AAV]).

摘要

最近,在器官离体灌流方面的进展使得实体器官在移植前可以有更长的保存时间,从而有可能在保存期间使供体器官复苏。在此延长的保存期间,有机会为整个心脏移植物提供病毒载体介导的基因治疗,这可能会改善心脏移植的结果。在这里,我们描述了如何通过常温离体灌流成功地将病毒载体递送到整个心脏移植物,以实现基因传递。该方案已通过基因治疗临床研究中最常用的病毒载体类型(腺病毒[Ad]和腺相关病毒载体[AAV])得到证实。

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