Genome Engineering for Personalized Arthritis Therapeutics.

Journal Article (Review)

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.

Full Text

Duke Authors

Cited Authors

  • Adkar, SS; Brunger, JM; Willard, VP; Wu, C-L; Gersbach, CA; Guilak, F

Published Date

  • October 2017

Published In

Volume / Issue

  • 23 / 10

Start / End Page

  • 917 - 931

PubMed ID

  • 28887050

Electronic International Standard Serial Number (EISSN)

  • 1471-499X

International Standard Serial Number (ISSN)

  • 1471-4914

Digital Object Identifier (DOI)

  • 10.1016/j.molmed.2017.08.002

Language

  • eng