What is the role of gene therapies in the treatment of dilated cardiomyopathy?

Updated: Mar 02, 2021
  • Author: Vinh Q Nguyen, MD, FACC; Chief Editor: Gyanendra K Sharma, MD, FACC, FASE  more...
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Early animal studies using recombinant adeno-associated viral gene therapy with gene transfer of phospholamban prevented deterioration of left ventricular systolic and diastolic function in genetically predisposed animals.

The use of vascular endothelial growth factor may have beneficial effects in persons with ischemic cardiomyopathies. This form of gene therapy has demonstrated the benefits of reducing revascularization and improving angina and quality of life.

The CUPID-2 trial (calcium upregulation by percutaneous administration of gene therapy in patients with cardiac disease) assessed the effectiveness of a gene transfer vector on the basis of adeno-associated virus 1 (AAV1)'s ability to deliver SERCA2a (sarcoplasmic/endoplasmic reticulum Ca(2+)-ATPase)–complementary DNA in patients with heart failure and reduced ejection fraction (LVEF < 35%). [143] Compared to placebo, there were no differences in recurrent heart failure-related hospitalizations or ambulatory treatment for heart failure, as well as no difference in the rate of death, heart transplantation, or mechanical circulatory support implantation. [143]

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