Cell therapy and gene therapy targeting GJB2 associated hearing loss

2015 Fiscal Year Final Research Report

• Project/Area Number: 25293351
• Research Category: Grant-in-Aid for Scientific Research (B)
• Allocation Type: Partial Multi-year Fund
• Section: 一般
• Research Field: Otorhinolaryngology
• Research Institution: Juntendo University
• Principal Investigator: Ikeda Katsuhisa 順天堂大学, 医学部, 教授 (70159614)
• Co-Investigator(Kenkyū-buntansha): MINOWA Osamu 理化学研究所, 疾患モデル評価研究開発チーム, 開発研究員 (00181967) ; KAMIYA Kazusaku 順天堂大学, 医学部, 准教授 (10374159) ; IIZUKA Takashi 順天堂大学, 医学部, 助教 (40372932)
• Project Period (FY): 2013-04-01 – 2016-03-31
• Keywords: 遺伝性難聴 / 遺伝子治療

Outline of Final Research Achievements

GJB2 encodes connexin (Cx) 26, a component in cochlear gap junction. We recently demonstrated that the drastic disruption of gap junction plaque (GJP) macromolecular complex composed of Cx26 and Cx30 are critical pathogenesis starting before hearing onset. To develop the effective therapy for GJB2 associated hearing loss, restoration of gap junction plaque (GJP) macromolecular complex using virus vectors or multipotent stem cells such as induced pluripotent stem (iPS) cells and mescenchymal stem cell (MSC) are expected to rescue the hearing function of GJB2 related hearing loss. Mouse induced pluripotent stem cells (iPS) were used for generation of Cx26-expressing cells with proper gap junction plaque between the cells. Adeno associate virus (AAV) were used for the GJB2 gene transfer and restoration of GJP. By differentiation of iPS cells, we generated the Cx26-expressiong cells with large gap junction plaque as cochlear cells.

Free Research Field

耳鼻咽喉科学