Combining iPS cell-derived myogenic progenitors and human artificial chromosomes for genomic-integration-free, systemically deliverable cell and gene therapy of Duchenne muscular dystrophy

G. Ferrari, L. M. Moyle,H. Hoshiya,M. Ragazzi,S. M. Maffioletti, T. Casteels,Y. Kazuki, F. Muntoni, M. Oshimura,F. S. Tedesco

HUMAN GENE THERAPY(2018)

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