Generating patient-specific induced pluripotent stem cells-derived cardiomyocytes for the treatment of cardiac diseases

Expert Opin Biol Ther. 2015;15(10):1399-409. doi: 10.1517/14712598.2015.1064109. Epub 2015 Jul 2.


Introduction: Induced pluripotent stem cells (iPSC) represent an appealing cell source to develop disease-modeling assays, drug testing assays and cell-based replacement therapies especially for cardiac disorders.

Areas covered: The development of efficient protocols to generate pure populations of cardiac myocytes is a prerequisite to provide reproducible, robust and valid assays. Different techniques have been recently proposed that allow production of high-yield high-quality cardiomyocytes. In addition, the newly developed genome-editing techniques offer multiple opportunities to manipulate the genome of patient-specific iPSC thus generating syngeneic iPSC lines. Genome-editing techniques will also allow engineering of iPSC to make them suitable for replacement therapies.

Expert opinion: Since their discovery, iPSCs have shown promise to revolutionize the way human diseases are studied. During the last years, different protocols have been developed to achieve reproducible and efficient differentiation of iPSCs including in cardiac and vascular cells. The recent introduction of the genome-editing techniques now allow targeted manipulation of the genome of patient-specific and control iPSCs lines. This approach would help to address a couple of current limitations, including the generation of isogenic lines for disease modeling and of clinical-grade lines for replacement therapy.

Keywords: disease models; drug testing; genome editing; pluripotent stem cells.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Cell Differentiation
  • Clustered Regularly Interspaced Short Palindromic Repeats / genetics
  • Heart Diseases / therapy*
  • Humans
  • Induced Pluripotent Stem Cells / cytology*
  • Myocytes, Cardiac / cytology
  • Myocytes, Cardiac / transplantation*