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Genome Engineering for Personalized Arthritis Therapeutics
Trends in Molecular Medicine ( IF 13.6 ) Pub Date : 2017-09-05 , DOI: 10.1016/j.molmed.2017.08.002
Shaunak S Adkar 1 , Jonathan M Brunger 2 , Vincent P Willard 3 , Chia-Lung Wu 4 , Charles A Gersbach 2 , Farshid Guilak 5
Affiliation  

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.



中文翻译:

个性化关节炎治疗的基因组工程

关节炎代表了一系列复杂的关节病理,导致大多数肌肉骨骼疾病。该家族中的几乎所有疾病,包括骨关节炎、类风湿性关节炎和幼年特发性关节炎,都是慢性疾病,很少或没有可用的疾病缓解疗法。基因组工程技术的进步,尤其是最近的 CRISPR-Cas9,彻底改变了我们询问和验证与关节炎等慢性疾病相关的遗传和表观遗传元件的能力。这些技术与细胞重编程方法(包括诱导多能干细胞的使用)一起,为人类疾病建模提供了一个平台。我们总结了来自全基因组关联研究和基因组学的新证据,证实了关节炎发病机制的遗传基础。我们还回顾了基因组工程在开发新的关节炎疗法中的潜在贡献。

更新日期:2017-09-05
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