GLI2-targeted gene therapy for musculoskeletal tumors

2014 Fiscal Year Final Research Report

• Project/Area Number: 24592237
• Research Category: Grant-in-Aid for Scientific Research (C)
• Allocation Type: Multi-year Fund
• Section: 一般
• Research Field: Orthopaedic surgery
• Research Institution: Kagoshima University
• Principal Investigator: NAGANO Satoshi 鹿児島大学, 医歯(薬)学総合研究科, 講師 (50373139)
• Co-Investigator(Kenkyū-buntansha): KOMIYA Setsuro 鹿児島大学, 医歯学総合研究科, 教授 (30178371) ; SETOGUCHI Takao 鹿児島大学, 医歯学総合研究科, 特任准教授 (40423727) ; KOSAI Kenichiro 鹿児島大学, 医歯学総合研究科, 教授 (90258418)
• Project Period (FY): 2012-04-01 – 2015-03-31
• Keywords: 遺伝子治療 / ヘッジホッグシグナル / 骨軟部腫瘍

Outline of Final Research Achievements

We showed arsenic trioxide targets GLI2, a crucial factor for intracellular signaling of musculoskeletal tumors, and suppressed growth of osteosarcoma cells. ATO is already approved for leukemia, therefore translation into clinical usage for osteosarcoma is anticipated. We also showed that Surv.m-CRA, a survivin-responsive conditionally-replicative adenovirus (CRA), exhibit strong antitumor activity to rhabdomyosarcoma stem cells. We found that ribosomal protein S3 is a target gene of GLI2 and its knockdown suppressed invasion and motility of osteosarcoma cells. We are preparing for clinical trial of Surv.m-CRA for musculoskeletal tumor patients, which is expected to further advance the research of gene therapy for musculoskeletal tumors.

Free Research Field

骨軟部腫瘍