Development of in vivo mRNA delivery system for treating diseases

2014 Fiscal Year Final Research Report

• Project/Area Number: 24300170
• Research Category: Grant-in-Aid for Scientific Research (B)
• Allocation Type: Partial Multi-year Fund
• Section: 一般
• Research Field: Biomedical engineering/Biological material science
• Research Institution: The University of Tokyo
• Principal Investigator: ITAKA Keiji 東京大学, 医学(系)研究科(研究院), 准教授 (60292926)
• Co-Investigator(Kenkyū-buntansha): OGATA Toru , 国立障害者リハビリテーションセンター(研究所), 研究部長 (00392192) ; KAWAGUCHI Hiroshi 東京大学, 医学部附属病院, 届出診療医 (40282660) ; ISHII Takehiko 東京大学, 大学院工学系研究科, 特任准教授 (80415075)
• Project Period (FY): 2012-04-01 – 2015-03-31
• Keywords: mRNA / DDS / ナノミセル / 神経障害

Outline of Final Research Achievements

The purpose of this study is to develop DDS for in vivo mRNA delivery and its application for treating animal disease models. By sophisticated molecular design of the cationic polymers for mRNA-loaded carriers, the parameters such as endosomal escape capacity and the stability under physiological conditions were well regulated, leading to development of protein expression-controllable system from mRNA. By introducing the mRNA-loaded carriers into subarachnoid space, the protein expression was obtained in a sustained manner for nearly a week. By administering BDNF-encoded mRNA using the carriers for model mice showing olfactory dysfunction, enhanced neurological recovery of olfactory function was achieved along with repairing the olfactory epithelium to a nearly normal architecture.

Free Research Field

DDS