Development of a therapeutic platform for the treatment of craniosynostosis using exosome-delivered siRNA.

2021 Fiscal Year Final Research Report

• Project/Area Number: 19K10400
• Research Category: Grant-in-Aid for Scientific Research (C)
• Allocation Type: Multi-year Fund
• Section: 一般
• Review Section: Basic Section 57070:Developmental dentistry-related
• Research Institution: Tokyo Medical and Dental University
• Principal Investigator: KOBAYASHI YUKIHO 東京医科歯科大学, 大学院医歯学総合研究科, 助教 (20596233)
• Project Period (FY): 2019-04-01 – 2022-03-31
• Keywords: 頭蓋縫合部早期癒合症

Outline of Final Research Achievements

To construct Ex-siRNAFgfr2S252W, exosomes were isolated and purified from mouse serum using an exosome isolation kit, and siRNA specific for the mouse Fgfr2 point mutation was introduced into the exosomes using the sRNA-into-Exome kit. The craniofacial sutures of embryonic wild-type mice and Apert syndrome model mice developing craniosynostosis were harvested at each developmental stage, and osteoblasts were isolated from the tissues and cultured. The mRNA and MAPK pathway factor phosphorylation that were specifically up-regulated and down-regulated in the cranial coronal sutures of Apert disease model mice were analyzed.

Free Research Field

歯科矯正学

Academic Significance and Societal Importance of the Research Achievements

得られる結果は、広く先天性疾患を対象としたsiRNA創薬の発展へと波及することが期待される。