High-Throughput Screening for Novel Drug Discovery Using Patient-Specific Induced Pluripotent Stem Cells for Familial Hypertrophic Cardiomyopathy

Abstract

The objective of this proposal is to discover novel drugs to treat hypertrophic cardiomyopathy (HCM) using cardiomyocytes (CMs) derived from human induced pluripotent stem cells (hiPSCs). HCM is one of the most prevalent heritable heart diseases in the world, affecting about 1 out of 500 people, including military families. It is characterized by a thickening of the heart tissue, reduced cavity size, impaired relaxation time, arrhythmias and ultimately sudden cardiac death (SCD). Here, we have validated drug candidates that lead to remodeling sarcomere and HCM disease progression in patient derived hiPSC-CMs. Additionally, we employed multiple HCM iPSC-CM lines to validate our findings to find novel drugs for multiple variants of HCM its underlying mechanisms.

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Document Details

Document Type
Technical Report
Publication Date
Jan 01, 2021
Accession Number
AD1133013

Entities

People

  • Jin‐Kyu Park

Organizations

  • Yale University

Tags

DTIC Thesaurus Topics

  • Abstracts
  • Biomedical Research
  • Cardiomyopathies
  • Cell Size
  • Cells
  • Department Of Defense
  • Disease Attributes
  • Diseases And Disorders
  • Electronic Mail
  • Families (Human)
  • Genes
  • Genetics
  • Heart
  • Heart Diseases
  • Maryland
  • Medical Personnel
  • Military Families
  • Mutations
  • Relaxation Time
  • Rna Sequence Analysis
  • Small Molecules
  • Stem Cells
  • Students
  • Therapy
  • Throughput

Fields of Study

  • Biology
  • Medicine

Readers

  • Cardiovascular Physiology
  • Molecular and Cellular Biology

Technology Areas

  • Biotechnology