Vascular Organoids to Model Inherited Vascular Diseases

Abstract

Hereditary Hemorrhagic Telangiectasia (HHT) is a debilitating condition characterized by vascular malformations and pulmonary hypertension (PH). Mutations in the ALK1 gene are a major cause of HHT-associated PH, but the underlying cellular and molecular mechanisms are poorly understood. To address this knowledge gap, this proposal aims to generate biomimetic vascular organoids from induced pluripotent stem cells (iPSCs) harboring two common ALK1 mutations. These organoids could provide a more realistic and physiologically relevant platform for studying the process of vascular remodeling (VAR) in HHT associated PH. This information could lead to the development of new therapeutic strategies for this devastating disease.

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Document Details

Document Type
Technical Report
Publication Date
Sep 01, 2023
Accession Number
AD1212040

Entities

People

  • Maria Trivieri

Organizations

  • Icahn School of Medicine at Mount Sinai

Tags

DTIC Thesaurus Topics

  • Biomedical Research
  • Blood Transfusions
  • Cardiovascular Physiological Phenomena
  • Cardiovascular System
  • Cell Line
  • Cells
  • Diseases
  • Endothelial Cells
  • Epithelial Cells
  • Genetic Engineering
  • Genetics
  • Pulmonary Hypertension
  • Small Molecules
  • Stem Cells
  • Three Dimensional
  • Vascular Diseases
  • Vascular Malformations
  • Vitamin C

Fields of Study

  • Biology

Readers

  • Cardiovascular Physiology
  • Molecular and Cellular Biology

Technology Areas

  • Biotechnology