Pferdehirt Lara, Damato Anna R, Lenz Kristin L, Gonzalez-Aponte Maria F, Palmer Daniel, Meng Qing-Jun, Herzog Erik D, Guilak Farshid
Department of Orthopedic Surgery, Washington University School of Medicine, St. Louis, MO, USA.
Shriners Hospitals for Children - Saint Louis, St. Louis, MO, USA.
Nat Commun. 2025 Feb 7;16(1):1457. doi: 10.1038/s41467-025-56584-5.
Circadian medicine, the delivery of therapeutic interventions based on an individual's daily rhythms, has shown improved efficacy and reduced side-effects for various treatments. Rheumatoid arthritis and other inflammatory diseases are characterized by diurnal changes in cytokines, leading to inflammatory flares, with peak disease activity in the early morning. Using a combination of synthetic biology and tissue engineering, we developed circadian-based gene circuits, termed "chronogenetics", that express a prescribed transgene downstream of the core clock gene promoter, Period2 (Per2). Gene circuits were transduced into induced pluripotent stem cells that were tissue-engineered into cartilage constructs. Our anti-inflammatory chronogenetic constructs produced therapeutic concentrations of interleukin-1 receptor antagonist in vitro. Once implanted in vivo, the constructs expressed circadian rhythms and entrained to daily light cycles, producing daily increases in biologic drug at the peak of Per2 expression. This approach represents the development of a cell-based chronogenetic therapy for various applications in circadian medicine.
昼夜节律医学,即基于个体日常节律提供治疗干预措施,已显示出在各种治疗中疗效提高且副作用减少。类风湿性关节炎和其他炎症性疾病的特征是细胞因子的昼夜变化,导致炎症发作,疾病活动在清晨达到高峰。通过结合合成生物学和组织工程,我们开发了基于昼夜节律的基因回路,称为“时辰遗传学”,它在核心时钟基因启动子Period2(Per2)的下游表达指定的转基因。基因回路被转导到诱导多能干细胞中,这些干细胞被组织工程化为软骨构建体。我们的抗炎时辰遗传学构建体在体外产生治疗浓度的白细胞介素-1受体拮抗剂。一旦植入体内,构建体就会表达昼夜节律并与每日光照周期同步,在Per2表达高峰时使生物药物每日增加。这种方法代表了一种基于细胞的时辰遗传学疗法的发展,可用于昼夜节律医学的各种应用。