Drug Discovery for Duchenne Muscular Dystrophy Using Patient-Derived Human iPSCs

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Grant Application Details

Application Title:

Drug Discovery for Duchenne Muscular Dystrophy Using Patient-Derived Human iPSCs

Public Abstract:
Research Objective

We will utilize human induced pluripotent stem cells derived from Duchenne muscular dystrophy (DMD) patients for drug testing and drug discovery for this rare genetic disease.


Diverse iPSC lines that recapitulate patient phenotypes will supplement preclinical studies to de-risk clinical trials while identifying a therapeutic target for DMD-associated cardiomyopathy.

Major Proposed Activities

  • Clinical evaluation of DMD patients recruited for the generation of iPSCs.
  • Generation of DMD induced pluripotent stem cell-derived cardiomyocytes (iPSC-CMs).
  • Cellular and molecular characterization of DMD iPSC-CMs.
  • Drug testing of GSB-D010 for safety and efficacy in DMD iPSC-CMs.
  • Drug screen of ~8,000 small molecules in DMD iPSC-CMs.
  • Validation of drug candidates in 3D cell models and in DMD mouse model.
Statement of Benefit to California:
Duchenne muscular dystrophy (DMD) is a genetic disorder affecting 1 in 3,500 male births and thus thousands are estimated to be affected in California. In DMD, cardiomyopathies are highly prevalent and the leading cause of death in the disease. By discovering a safe and effective drug for these heart problems, we can help meet an urgent need for DMD patients and establish a proof of concept in applying induced pluripotent stem cell technology for the discovery of drugs for rare orphan diseases.