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Article

Autologous Cell Therapy Approach for Duchenne Muscular Dystrophy using PiggyBac Transposons and Mesoangioblasts.

Molecular therapy : the journal of the American Society of Gene Therapy - 4 Apr 2018

Iyer Pavithra S, Mavoungou Lionel O, Ronzoni Flavio, Zemla Joanna, Schmid-Siegert Emanuel, Antonini Stefania, Neff Laurence A, Dorchies Olivier M, Jaconi Marisa, Lekka Malgorzata, Messina Graziella, Mermod Nicolas

Abstract excerpt

Duchenne muscular dystrophy (DMD) is a lethal muscle-wasting disease currently without cure. We investigated the use of the PiggyBac transposon for full-length dystrophin expression in murine mesoangioblast (MABs) progenitor cells. DMD murine MABs were transfected with transposable expression vectors for full-length dystrophin and transplanted intramuscularly or intra-arterially into mdx/SCID mice. Intra-arterial...

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Source

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Identifiers

PMID: 29503200
DOI: 10.1016/j.ymthe.2018.01.021
PMCID: PMC6079556

Genes

DMD

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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