Research and Development Projects Adopted in FY2015
Development of a novel muscle-directed DDS for peptide-conjugated morpholino
Project Leader:Okada Takashi
Professor, Graduate School of Medicine, Nippon Medical School
A considerable amount of attention has been focused on the oligonucleotide-based exon-skipping therapy to restore dystrophin expression in the affected muscle tissues of Duchenne muscular dystrophy patients with promising data obtained from a series of world-wide clinical trials. One of the current concerns in the oligonucleotide-based approach is the limited efficacy in the skeletal muscles including diaphragm due to a lack of the adequate drug delivery system (DDS).
To address this issue, we have recently demonstrated an innovative work published in the Nano Letters (Ezzat and Aoki et al. 2015) to state that self-assembly of nanoparticles is essential for receptor-mediated uptake of the peptide-conjugated morpholino antisense oligo (P-PMO). In addition, we also successfully established a large-scale manufacturing methods for the muscle-directed AAV type 8 (AAV8) empty capsids as a candidate of a novel DDS (Kinoh and Okada et al., unpublished).
