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Genome Engineering for Personalized Arthritis Therapeutics
Shaunak S Adkar1, Jonathan M Brunger2, Vincent P Willard3
1Department of Orthopedic Surgery, Washington University, St. Louis, MO 63110, USA; Shriners Hospitals for Children - St. Louis, St. Louis, MO 63110, USA; Department of Cell Biology, Duke University Medical Center, Durham, NC 27710, USA.
Trends in Molecular Medicine
|September 10, 2017
Summary
Genome engineering and stem cell technologies offer new hope for arthritis treatment. These tools help model arthritis and identify genetic targets for developing novel disease-modifying therapies.
Area of Science:
- Genetics and genomics
- Molecular biology
- Immunology
Background:
- Arthritis encompasses numerous chronic joint diseases like osteoarthritis and rheumatoid arthritis.
- Current treatments for arthritis lack disease-modifying capabilities.
- Genetic and epigenetic factors are implicated in arthritis pathogenesis.
Purpose of the Study:
- To review the genetic basis of arthritis.
- To explore the role of genome engineering in arthritis research.
- To discuss the potential of new technologies for arthritis therapeutics.
Main Methods:
- Review of genome-wide association studies (GWAS) and genomics data.
- Analysis of advances in genome engineering, including CRISPR-Cas9.
- Evaluation of cell reprogramming methods like induced pluripotent stem cells (iPSCs) for disease modeling.
Main Results:
- Genomic studies confirm a significant genetic contribution to arthritis development.
- Genome engineering tools enable precise interrogation of genetic elements in arthritis.
- iPSCs provide a platform for creating humanized models of arthritis.
Conclusions:
- Genetic insights are crucial for understanding arthritis.
- Genome engineering and iPSC technology hold promise for developing novel arthritis therapeutics.
- Future research should focus on translating these technologies into clinical applications for arthritis.
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