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

Authors
 Yun-Rak Choi  ;  Kelsey H. Collins  ;  Jin-Woo Lee  ;  Ho-Jung Kang  ;  Farshid Guilak 
Citation
 TISSUE ENGINEERING AND REGENERATIVE MEDICINE, Vol.16(4) : 335-343, 2019 
Journal Title
TISSUE ENGINEERING AND REGENERATIVE MEDICINE
ISSN
 1738-2696 
Issue Date
2019
Keywords
Gene editing ; Genetics ; Osteoarthritis ; Personalized medicine
Abstract
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.
Full Text
https://link.springer.com/article/10.1007/s13770-018-0172-4
DOI
10.1007/s13770-018-0172-4
Appears in Collections:
1. College of Medicine (의과대학) > Dept. of Orthopedic Surgery (정형외과학교실) > 1. Journal Papers
Yonsei Authors
Kang, Ho Jung(강호정) ORCID logo https://orcid.org/0000-0003-0273-1264
Lee, Jin Woo(이진우) ORCID logo https://orcid.org/0000-0002-0293-9017
Choi, Yun Rak(최윤락)
URI
https://ir.ymlib.yonsei.ac.kr/handle/22282913/173377
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