Genome Engineering for Personalized Arthritis Therapeutics.

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.

journal_name

Trends Mol Med

authors

Adkar SS,Brunger JM,Willard VP,Wu CL,Gersbach CA,Guilak F

doi

10.1016/j.molmed.2017.08.002

subject

Has Abstract

pub_date

2017-10-01 00:00:00

pages

917-931

issue

10

eissn

1471-4914

issn

1471-499X

pii

S1471-4914(17)30142-9

journal_volume

23

pub_type

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