Genome Engineering for Personalized Arthritis Therapeutics.

TitleGenome Engineering for Personalized Arthritis Therapeutics.
Publication TypeJournal Article
Year of Publication2017
AuthorsSS Adkar, JM Brunger, VP Willard, C-L Wu, CA Gersbach, and F Guilak
JournalTrends in Molecular Medicine
Volume23
Start Page917
Issue10
Pagination917 - 931
Date Published10/2017
Abstract

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.

DOI10.1016/j.molmed.2017.08.002
Short TitleTrends in Molecular Medicine