Stem Cell Therapy in Single-Ventricle Physiology: Recent Progress and Future Directions

Semin Thorac Cardiovasc Surg Pediatr Card Surg Annu. 2021:24:67-76. doi: 10.1053/j.pcsu.2021.03.003.


Current surgical and medical treatment options for single ventricle physiology conditions remain palliative. On the long term, despite treatment, the systemic ventricle has a significant risk of developing failure. There are unmet needs to develop novel treatment modalities to help ameliorate the ventricular dysfunction. Advances in the field of stem cell therapy have been promising for the treatment of heart failure. Numerous stem cell populations have been identified. Preclinical studies in small and large animal models provide evidence for effectiveness of this treatment modality and reveal several mechanisms of action by which stem cells exert their effect. Many clinical trials have been designed to further investigate the therapeutic potential that stem cell therapy may hold for pediatric populations with single ventricle physiology. In this review, we discuss the stem cell types used in these populations, some preclinical studies, and the clinical trials of stem cell therapy in single ventricle patients.

Keywords: Cavopulmonary shunt; Fontan circulation; Heart failure; Right ventricle; Single ventricle; Stem cells; Systemic ventricle.

Publication types

  • Review

MeSH terms

  • Child
  • Clinical Trials as Topic
  • Fontan Procedure*
  • Heart Defects, Congenital* / therapy
  • Heart Failure* / therapy
  • Heart Ventricles
  • Humans
  • Stem Cell Transplantation
  • Univentricular Heart*