Gene therapy in cardiovascular diseases: A review of recent updates

Abstract

Gene therapy is considered as a promising approach for treating cardiac dysfunction. In this review, we evaluated the clinical trials assessing gene therapy in cardiovascular diseases (CVD) from 2000 to 2017. PubMed and ClinicalTrials.gov (only English language) were searched for clinical trials published between January 2000 and May 2017, using the search terms "gene transfer" OR "gene therapy" and "cardiovascular diseases" and related terms. The trials with sample size lower than 10 patients were excluded. Twenty-six clinical trials on human and animals, including 1543 patients were listed and evaluated. The sample size in 14 trials was lower than 100 patients and in seven trials lower than 20 patients. Eleven trials used plasmid DNA and eight trials used adenovirus, one study used plasmid DNA, adenovirus, and liposome. We detected that gene therapy was a safe approach and improved the symptoms of CVD; however, the effect of gene therapy on the cardiac dysfunction is controversial.

Recombinant Adenovirus siRNA Knocking Down the Ndufs4 Gene Alleviates Myocardial Apoptosis Induced by Oxidative Stress Injury

“…We also verifed Ndufs4 siRNA efectiveness in H9c2 cells by Western blot and realtime PCR, the Ndufs4 siRNA reduced the expression of mRNA and protein of the Ndufs4 gene by 76.7% and 64.9%, respectively. In recent years, gene therapy has emerged as a promising therapeutic avenue for the therapy of some diseases, such as cancer or cardiovascular disease [25][26][27][28]. Owing to specifc gene silencing, siRNA is expected to become an approach in treating some diseases [29].…”

“…In recent years, gene therapy has emerged as a promising therapeutic avenue for the therapy for cancer [24] and even heart disease [25], with numerous studies reporting its positive efects on cardiac function in cultured cells or small animal models. Adenovirus is the most widely used vector for its merits of broad host range, rapid propagation period, high infection rate, and convenient storage [26].…”

Gene Therapy of Chronic Limb-Threatening Ischemia: Vascular Medical Perspectives

“…By the fusion of liposomes with the cell wall of the target cells, the introduced substance in the liposome may be released into the cytosol and act there either directly or, in the case of DNA, initiate the production of corresponding proteins. As a vehicle for the therapeutic genes, plasmids are often used [ 63 , 64 , 65 , 66 , 67 , 68 , 69 , 70 , 71 , 72 , 73 , 74 , 75 , 76 , 77 ]. Plasmids are circular DNA, existing as extrachromosomal DNA in the cytosol and having a cell-independent replication cycle.…”