Department of Genetics and Genomic Sciences, Icahn Institute for Genomics and Multiscale Biology, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
Department of Cardiovascular Surgery and Pediatric Cardiac Surgery, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
Methods Mol Biol. 2022;2573:135-145. doi: 10.1007/978-1-0716-2707-5_10.
Gene therapy is a promising approach in the treatment of cardiovascular diseases. The vectors available for cardiovascular gene therapy have significantly improved over time. Cardiac tropism is a primary characteristic of an ideal vector along with a long-term expression profile and a minimal risk of cellular immune response. Preclinical and clinical studies have demonstrated that adeno-associated viral (AAV) vectors are one of the most attractive vehicles for gene transfer. AAV has gained great popularity in the last years because of its biological properties and advantages over other viral vector systems. In this chapter we will describe methods for intracardiac delivery of AAV vector in rats.
基因治疗是治疗心血管疾病的一种很有前途的方法。用于心血管基因治疗的载体随着时间的推移有了显著的改善。心脏趋向性是理想载体的主要特征之一，还需要具有长期表达谱和最小的细胞免疫反应风险。临床前和临床研究表明，腺相关病毒（AAV）载体是基因转移最有吸引力的载体之一。AAV 因其生物学特性和优于其他病毒载体系统的优势，近年来受到了广泛关注。在本章中，我们将描述在大鼠中心内递送 AAV 载体的方法。
