Research in iPSC-Based Disease Models
A special issue of International Journal of Molecular Sciences (ISSN 1422-0067). This special issue belongs to the section "Molecular Biology".
Deadline for manuscript submissions: 20 December 2024 | Viewed by 1473
Special Issue Editor
Special Issue Information
Dear Colleagues,
Induced pluripotent stem cells (iPSCs) represent a new disease model and drug discovery tool, challenging the current limitations of animal models. Patient-specific iPSCs provide unlimited access to disease-relevant cells that are difficult to harvest, such as brain neurons and cardiomyocytes. Phenotypes associated with various diseases can be monitored and tested by using diverse cell types differentiated from iPSCs in culture. Monolayer 2D iPSC-derived cultures are convenient for the high-throughput screening of new drugs, some of which are being tested in clinical trials; however, 3D organoid models better reflect the cell–matrix interaction found in tissues and organs in vivo, such as the brain, heart, liver, kidney, gut, and lung. For example, midbrain organoids can model the key features of Parkinson’s disease. Moreover, the emergent gene editing fields provide the capacity to introduce or correct disease mutations in iPSCs to assess their contributions. Editing disease mutations in iPSCs to generate isogenic controls can shed light onto the causal relationship between genotypes and phenotypes; therefore, iPSCs have emerged as a wonderful model to facilitate new drug discovery and drug repositioning in human health.
This Special Issue will include recent advances in iPSC-based models of various diseases, in which gene editing is leveraged to highlight the impact of risk mutations.
Dr. Eric Deneault
Guest Editor
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Keywords
- iPSCs
- disease model
- organoid
- gene editing
- mutation correction
- isogenic control
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