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Updated: Jan 20, 2026
Mouse Genome Engineering Using Designer Nucleases
Published on: April 2, 2014
Genome Engineering for Osteoarthritis: From Designer Cells to Disease-Modifying Drugs
Yun-Rak Choi1,2,3, Kelsey H Collins1,2, Jin-Woo Lee3
11Department of Orthopaedic Surgery, Washington University in St. Louis, 1 Brookings Dr, St. Louis, MO 63130 USA.
Background:
Osteoarthritis (OA) is a highly prevalent degenerative joint disease involving joint cartilage and its surrounding tissues. OA is the leading cause of pain and disability worldwide. At present, there are no disease-modifying OA drugs, and the primary therapies include exercise and nonsteroidal anti-inflammatory drugs until total joint replacement at the end-stage of the disease.
Methods:
In this review, we summarized the current state of knowledge in genetic and epigenetic associations and risk factors for OA and their potential diagnostic and therapeutic applications.
Results:
Genome-wide association studies and analysis of epigenetic modifications (such as miRNA expression, DNA methylation and histone modifications) conducted across various populations support the notion that there is a genetic basis for certain subsets of OA pathogenesis.
Conclusion:
With recent advances in the development of genome editing technologies such as the CRISPR-Cas9 system, these genetic and epigenetic alternations in OA can be used as platforms from which potential biomarkers for the diagnosis, prognosis, drug response, and development of potential personalized therapeutic targets for OA can be approached. Furthermore, genome editing has allowed the development of "designer" cells, whereby the receptors, gene regulatory networks, or transgenes can be modified as a basis for new cell-based therapies.
Insights
Genetic and epigenetic factors are key to osteoarthritis (OA) development. Genome editing offers new avenues for diagnosing and treating this widespread joint disease.
Area of Science:
- Genetics and Epigenetics
- Osteoarthritis Research
Background:
- Osteoarthritis (OA) is a prevalent degenerative joint disease causing global pain and disability.
- Current OA treatments focus on symptom management and joint replacement, lacking disease-modifying drugs.
Purpose of the Study:
- To review genetic and epigenetic factors in OA.
- To explore potential diagnostic and therapeutic applications of these factors.
Main Methods:
- Review of genome-wide association studies (GWAS).
- Analysis of epigenetic modifications including miRNA expression, DNA methylation, and histone modifications.
Main Results:
- Evidence supports a genetic basis for subsets of OA pathogenesis.
- Genetic and epigenetic alterations are linked to OA development.
Conclusions:
- Genome editing technologies like CRISPR-Cas9 enable biomarker discovery for OA diagnosis and prognosis.
- These advancements facilitate personalized therapeutic targets and novel cell-based therapies for OA.
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