心血管疾病的基因治疗

doi:10.3969/j.issn.1007-2861.2016.03.013

Abstract

Abstract:

Gene therapy shows great promise in the treatment of both inherited and acquired cardiovascular diseases. Identification of key molecule players in pathophysiology of cardiovascular disease and development of human disease genomic research lead to encouraging preclinical gene therapy studies in animal models. However, the presence of cellular immune responses, insufficient gene expression level and overall limited in vivo gene transduction efficiencies have hampered the translational progress to clinical use of gene therapy. In recent years, improvements in gene delivery system and discovery of advanced genome editing technologies open new therapeutic perspectives, with clustered regularly interspaced short palindromic repeats (CRISRP)/Cas9 genome editing technology already being successfully used in animal models to treat hypercholesterolemia. Further improvement in gene delivery efficiency, increase in targeting specificity of genome editing tools, and establishment of experimental systems for a thorough analysis of potential safety problems would help eventually bring gene therapy for heart disease to reality. In this review, recent advances in the use of different delivery vectors and CRISPR/Cas9 genome editing technology in gene therapy research to treat cardiovascular diseases are discussed.

Key words: CRISPR/Cas9 genome editing technology, gene delivery vector , gene therapy , cardiovascular disease

DING Qiurong, CHEN Yanhao. Gene therapy for cardiovascular disease[J]. Journal of Shanghai University（Natural Science Edition）, 2016, 22(3): 270-279.

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Gene therapy for cardiovascular disease

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