Genome Engineering for Osteoarthritis: From Designer Cells to Disease-Modifying Drugs

Yun Rak Choi, Kelsey H. Collins, Jin Woo Lee, Ho Jung Kang, Farshid Guilak

Research output: Contribution to journalReview articlepeer-review

14 Citations (Scopus)


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.

Original languageEnglish
Pages (from-to)335-343
Number of pages9
JournalTissue Engineering and Regenerative Medicine
Issue number4
Publication statusPublished - 2019 Aug 14

Bibliographical note

Funding Information:
We acknowledge that this review is not exhaustive and the authors apologize to those whose work was not included due to space limitations. This study was supported in part by NIH Grants AR50245, AR48852, AG15768, AR48182, AG46927, OD10707, DK108742, EB018266, AR057235, AR073752, the Arthritis Foundation, and the Nancy Taylor Foundation for Chronic Diseases.

Publisher Copyright:
© 2019, The Korean Tissue Engineering and Regenerative Medicine Society and Springer Nature B.V.

All Science Journal Classification (ASJC) codes

  • Medicine (miscellaneous)
  • Biomedical Engineering


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