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Module 7: Clinical Applications of Stem Cell Biology 

Introduction and Learning Objectives: 

Module 7 discusses how stem cell technologies can be leveraged for clinical applications. The module builds upon developmental biology concepts applied to stem cell differentiation, transdifferentiation, and direct reprogramming for therapeutic purposes. It reinforces the advantage of embryonic and adult stem cell technologies for in vitro and in vivo disease modeling. Specific examples are provided for stem cell applications, from using them to compare normal and pathological states to uncovering novel treatments to cell replacement therapies. Finally, students will be presented with an introduction to ethical, business, and legal frameworks related to stem cells.

At the conclusion of this module, students should be able to:

  • Define different methods for using stem cells to study and treat disease;
  • Distinguish disease modeling from cell replacement;
  • Compare the use of pluripotent or adult stem cell strategies to treat different disease scenarios;
  • Calculate potential risks of utilizing stem cells as a therapeutic strategy;
  • Formulate a workflow for how one would apply these techniques to a new disease;
  • Identify what tests must be completed before a new cell therapy can be considered for clinical application;
  • Debate whether there are some diseases for which cell replacement therapy would not work.

Core Concepts

  1. Overview of clinical application of stem cell technologies 
  2. Pluripotent stem cell differentiation
  3. Transdifferentiation and direct programming
  4. Adult stem cell disease modeling and therapy
  5. Stem cell therapy guidelines and clinical trials

This module is part of the ISSCR's Core Concepts in Stem Cell Biology: Syllabus and Learning Guide. To access the experiments, theories, and primary and secondary literature that support each of these concepts, download the complete resource by using the form on the right-hand side of this webpage.

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Education Committee

Esteban Orlando Mazzoni, PhD (Chair)
New York University, USA

William J. Anderson, PhD
Harvard University, USA

Sina Bartfeld, PhD
University of Wuerzburg, Germany

Anna Couturier
EuroStemCell and University of Edinburgh, UK

Yvanka de Soysa, PhD
Boston Children's Hospital, USA

Robin Scott Hawley, PhD
Stowers Institute for Medical Research, USA

Ping Hu, PhD
Shanghai Institute of Biochemistry and Cell Biology Chinese Academy of Sciences, China

Yuin Han Jonathan Loh, PhD
Institute of Molecular and Cell Biology, Singapore

Lolitika Mandal, PhD
Indian Institute of Science Education and Research Mohali, India

Zubin Master, PhD
Mayo Clinic, USA

Alysson Renato Muotri, PhD
University of California, San Diego, USA

Jose Maria Polo, PhD
Monash University, Australia

Julie Perlin, PhD
International Society for Stem Cell Research, USA