2013 Fiscal Year Final Research Report
Gene therapy for achodroplasia using a biocompatible non-viral gene delivery system
| Project/Area Number |
23659708
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| Research Category |
Grant-in-Aid for Challenging Exploratory Research
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| Allocation Type | Multi-year Fund |
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| Research Field |
Orthopaedic surgery
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| Research Institution | The University of Tokyo |
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Principal Investigator |
ITAKA Keiji 東京大学, 医学(系)研究科(研究院), 准教授 (60292926)
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| Co-Investigator(Renkei-kenkyūsha) |
HAGA Nobuhiko 東京大学, 医学部附属病院, 教授 (80251263)
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| Project Period (FY) |
2011 – 2013
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| Keywords | 軟骨無形成症 / 遺伝子治療 / 細胞治療 / 細胞移植 / 遺伝子キャリア / スフェロイド / C型ナトリウム利尿ペプチド |
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| Research Abstract |
This study is aiming at definitive therapy of achondroplasia by regulating FGFR3 signals using CNP (C-type natriuretic peptide)-expressing gene. Introducing the gene into wild-type infant mice using a non-viral gene carrier, polyplex nanomicelle, induced a therapeutic effect of bone elongation of the extremities at approximately 2 % compared with control mice. However, in experiments using model mice of achondroplasia, no significant effects were observed. Pursuing a more effective technique to introduce gene, cell spheroid transplantation system combined with genetic modification was investigated. Subcutaneous transplantation of spheroids that had received gene transfection using the nanomicelle provided high continuous transgene expression in host mice. We are planning to apply the cell transplantation system for treating achondroplasia in the future study.
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