Genome Engineering for Personalized Arthritis Therapeutics
| Autor: | Charles A. Gersbach, Shaunak S. Adkar, Jonathan M. Brunger, Farshid Guilak, Chia-Lung Wu, Vincent P. Willard |
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| Rok vydání: | 2017 |
| Předmět: |
0301 basic medicine
Arthritis Genomics Genome-wide association study Bioinformatics Article Genome engineering 03 medical and health sciences medicine Animals Humans Epigenetics Precision Medicine Induced pluripotent stem cell Molecular Biology Gene Editing Genome Human business.industry medicine.disease 030104 developmental biology Rheumatoid arthritis Molecular Medicine CRISPR-Cas Systems business Reprogramming Genome-Wide Association Study |
| Zdroj: | Trends in Molecular Medicine. 23:917-931 |
| ISSN: | 1471-4914 |
| Popis: | Arthritis represents a family of complex joint pathologies responsible for the majority of musculoskeletal conditions. Nearly all diseases within this family, including osteoarthritis, rheumatoid arthritis, and juvenile idiopathic arthritis, are chronic conditions with few or no disease-modifying therapeutics available. Advances in genome engineering technology, most recently with CRISPR-Cas9, have revolutionized our ability to interrogate and validate genetic and epigenetic elements associated with chronic diseases such as arthritis. These technologies, together with cell reprogramming methods, including the use of induced pluripotent stem cells, provide a platform for human disease modeling. We summarize new evidence from genome-wide association studies and genomics that substantiates a genetic basis for arthritis pathogenesis. We also review the potential contributions of genome engineering in the development of new arthritis therapeutics. |
| Databáze: | OpenAIRE |
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