Gene therapy for achodroplasia using a biocompatible non-viral gene delivery system

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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.