Related Experiment Video
Updated: Jul 8, 2026

09:03
Introducing Point Mutations into Human Pluripotent Stem Cells Using Seamless Genome Editing
Published on: May 10, 2020
From Gene Function to Precision Intervention: CRISPR/Cas9 and Stem Cell-Based Strategies as Emerging
Masuma Khatun1,2, Karolina Lundin3, Timo Tuuri3
1Department of Obstetrics and Gynecology, University of Helsinki and Helsinki University Hospital, Haartmaninkatu 2, 00290, Helsinki, Finland. masuma.khatun@helsinki.fi.
Stem Cell Reviews and Reports
|July 7, 2026
Summary
Polyendocrine metabolic ovarian syndrome (PMOS) is a complex disorder affecting women
Area of Science:
- Reproductive Endocrinology and Metabolism
- Genetics and Genomics
- Regenerative Medicine
Background:
- Polyendocrine metabolic ovarian syndrome (PMOS) affects 18% of women globally, causing anovulatory infertility.
- Current PMOS treatments manage symptoms but not underlying molecular disruptions.
- Multi-omic studies reveal genetic links and pathway disturbances in PMOS.
Purpose of the Study:
- To explore novel therapeutic strategies for PMOS by integrating functional genomics and stem cell research.
- To investigate the potential of CRISPR/Cas9 and stem cell-based platforms for PMOS.
- To shift PMOS management towards mechanism-driven interventions.
Main Methods:
- Utilizing CRISPR/Cas9 gene editing for precise pathway interrogation.
- Employing stem cell platforms: mesenchymal stem cells (MSCs), exosomes, and gene-edited induced pluripotent stem cells (iPSCs).
- Conducting preclinical studies on MSCs and iPSC-based models for disease modeling and regeneration.
Main Results:
- MSCs and derivatives show potential in modulating inflammation, restoring ovarian structure, and improving metabolic parameters.
- iPSC models facilitate patient-specific investigation of steroidogenic and metabolic abnormalities.
- CRISPR-based functional genomics combined with stem cell research offers a promising approach for PMOS.
Conclusions:
- Integrating CRISPR-based functional genomics with stem cell research represents a paradigm shift in PMOS management.
- This approach moves beyond symptom management towards targeted, mechanism-driven interventions.
- Future research should address translational challenges for effective PMOS treatment.
Related Concept Videos
CRISPR
Genome editing technologies allow scientists to modify an organism’s DNA via the addition, removal, or rearrangement of genetic material at specific genomic locations. These types of techniques could potentially be used to cure genetic disorders such as hemophilia and sickle cell anemia. One popular and widely used DNA-editing research tool that could lead to safe and effective cures for genetic disorders is the CRISPR-Cas9 system. CRISPR-Cas9 stands for Clustered Regularly Interspaced Short...
CRISPR/Cas9 Genome Editing
The CRISPR-Cas system serves as a bacterial defense mechanism against invading genetic elements such as viruses and plasmids, forming the foundation for its adaptation as a powerful genome-editing tool. Originally discovered in prokaryotes, this system has been repurposed to revolutionize genetic engineering across a wide range of organisms, including plants, animals, and humans. The core component, Cas9, is an endonuclease derived from Streptococcus pyogenes, capable of introducing...
