Abstract
Recent progress in the field of cellular reprogramming has opened up the doors to a new era of disease modelling, as pluripotent stem cells representing a myriad of genetic diseases can now be produced from patient tissue. These cells can be expanded and differentiated to produce a potentially limitless supply of the affected cell type, which can then be used as a tool to improve understanding of disease mechanisms and test therapeutic interventions. This process requires high levels of scrutiny and validation at every stage, but international standards for the characterisation of pluripotent cells and their progeny have yet to be established. Here we discuss the current state of the art with regard to modelling diseases affecting the ectodermal, mesodermal and endodermal lineages, focussing on studies which have demonstrated a disease phenotype in the tissue of interest. We also discuss the utility of pluripotent cell technology for the modelling of cancer and infectious disease. Finally, we spell out the technical and scientific challenges which must be addressed if the field is to deliver on its potential and produce improved patient outcomes in the clinic.
Keywords: Induced pluripotent stem cells, human embryonic stem cells, neurodevelopmental disorders, endodermal disorders, mesodermal disorders, reprogramming, disease modelling
Current Gene Therapy
Title:Modelling Human Disease with Pluripotent Stem Cells
Volume: 13 Issue: 2
Author(s): Richard Siller, Sebastian Greenhough, In-Hyun Park and Gareth J. Sullivan
Affiliation:
Keywords: Induced pluripotent stem cells, human embryonic stem cells, neurodevelopmental disorders, endodermal disorders, mesodermal disorders, reprogramming, disease modelling
Abstract: Recent progress in the field of cellular reprogramming has opened up the doors to a new era of disease modelling, as pluripotent stem cells representing a myriad of genetic diseases can now be produced from patient tissue. These cells can be expanded and differentiated to produce a potentially limitless supply of the affected cell type, which can then be used as a tool to improve understanding of disease mechanisms and test therapeutic interventions. This process requires high levels of scrutiny and validation at every stage, but international standards for the characterisation of pluripotent cells and their progeny have yet to be established. Here we discuss the current state of the art with regard to modelling diseases affecting the ectodermal, mesodermal and endodermal lineages, focussing on studies which have demonstrated a disease phenotype in the tissue of interest. We also discuss the utility of pluripotent cell technology for the modelling of cancer and infectious disease. Finally, we spell out the technical and scientific challenges which must be addressed if the field is to deliver on its potential and produce improved patient outcomes in the clinic.
Export Options
About this article
Cite this article as:
Siller Richard, Greenhough Sebastian, Park In-Hyun and J. Sullivan Gareth, Modelling Human Disease with Pluripotent Stem Cells, Current Gene Therapy 2013; 13 (2) . https://dx.doi.org/10.2174/1566523211313020004
DOI https://dx.doi.org/10.2174/1566523211313020004 |
Print ISSN 1566-5232 |
Publisher Name Bentham Science Publisher |
Online ISSN 1875-5631 |
Call for Papers in Thematic Issues
Programmed Cell Death Genes in Oncology: Pioneering Therapeutic and Diagnostic Frontiers (BMS-CGT-2024-HT-45)
Programmed Cell Death (PCD) is recognized as a pivotal biological mechanism with far-reaching effects in the realm of cancer therapy. This complex process encompasses a variety of cell death modalities, including apoptosis, autophagic cell death, pyroptosis, and ferroptosis, each of which contributes to the intricate landscape of cancer development and ...read more
Related Journals
- Author Guidelines
- Graphical Abstracts
- Fabricating and Stating False Information
- Research Misconduct
- Post Publication Discussions and Corrections
- Publishing Ethics and Rectitude
- Increase Visibility of Your Article
- Archiving Policies
- Peer Review Workflow
- Order Your Article Before Print
- Promote Your Article
- Manuscript Transfer Facility
- Editorial Policies
- Allegations from Whistleblowers
- Announcements
Related Articles
-
Editorial (Hot Topic: Introduction to the Special Issue: Relevance of Endocrine and Metabolic Disorders in Heart Failure: From Pathophysiology to Therapeutic Approach)
Endocrine, Metabolic & Immune Disorders - Drug Targets Hypertension in Patients with Severe Aortic Stenosis: Emphasis on Antihypertensive Treatment and the Risk of Syncope
Current Hypertension Reviews Patent Selections
Recent Patents on Medical Imaging MiR-106a Associated with Diabetic Peripheral Neuropathy Through the Regulation of 12/15-LOX-meidiated Oxidative/Nitrative Stress
Current Neurovascular Research Conditional Cardiac Overexpression of S100A6 Attenuates Myocyte Hypertrophy and Apoptosis Following Myocardial Infarction
Current Pharmaceutical Design Physical Activity and Diabetic Cardiomyopathy: Myocardial Adaptation Depending on Exercise Load
Current Diabetes Reviews Neuroprotection by Resveratrol in Diabetic Neuropathy: Concepts & Mechanisms
Current Medicinal Chemistry The Potential Role of Erythropoietin as a Pleiotropic Agent in Post-cardiac Arrest Syndrome
Current Pharmaceutical Design Amino Acid and Polyamine Membrane Transporters in Trypanosoma cruzi: Biological Function and Evaluation as Drug Targets
Current Medicinal Chemistry Pompe Disease: From New Views on Pathophysiology to Innovative Therapeutic Strategies
Current Pharmaceutical Biotechnology Role of Peroxynitrite in the Cardiovascular Dysfunction of Septic Shock
Current Vascular Pharmacology Stem Cell Therapy in Chronic Ischemic Heart Dysfunction with and Without Viability
Cardiovascular & Hematological Disorders-Drug Targets Ignored Avenues in Alpha-Synuclein Associated Proteopathy
CNS & Neurological Disorders - Drug Targets N-acetyl-seryl-aspartyl-lysyl-proline (Ac-SDKP): Potential target molecule in research of heart, kidney and brain
Current Pharmaceutical Design Simultaneous Spectrophotometric Determination of Iron (III) and Vanadium (V) in Water Samples Using a Combination of Partial Least Squares Regression and Solid Phase Extraction with Modified Octadecyl Silica Membrane Disks
Current Analytical Chemistry Therapeutic Potential of Targeting Protease Activated Receptors in Cardiovascular Diseases
Current Pharmaceutical Design Interrelationship Between Kidney Function and Percutaneous Mitral Valve Interventions: A Comprehensive Review
Current Cardiology Reviews Inhibitory Effect of Fruit Juices on the Doxorubicin Metabolizing Activity of Carbonyl Reductase 1
Drug Metabolism Letters Role of Nitrosative Stress and Poly(ADP-ribose) Polymerase Activation in Diabetic Vascular Dysfunction
Current Vascular Pharmacology Nitric Oxide in Cardiac Transplantation
Vascular Disease Prevention (Discontinued)