Reversible immortalisation enables genetic correction of human muscle progenitors and engineering of next-generation human artificial chromosomes for Duchenne muscular dystrophy.

doi:10.15252/emmm.201607284

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Article

Reversible immortalisation enables genetic correction of human muscle progenitors and engineering of next-generation human artificial chromosomes for Duchenne muscular dystrophy.

EMBO molecular medicine - 1 Feb 2018

Benedetti Sara, Uno Narumi, Hoshiya Hidetoshi, Ragazzi Martina, Ferrari Giulia, Kazuki Yasuhiro, Moyle Louise Anne, Tonlorenzi Rossana, Lombardo Angelo, Chaouch Soraya, Mouly Vincent, Moore Marc, Popplewell Linda, Kazuki Kanako, Katoh Motonobu, Naldini Luigi, Dickson George, Messina Graziella, Oshimura Mitsuo, Cossu Giulio, Tedesco Francesco Saverio

Abstract excerpt

Transferring large or multiple genes into primary human stem/progenitor cells is challenged by restrictions in vector capacity, and this hurdle limits the success of gene therapy. A paradigm is Duchenne muscular dystrophy (DMD), an incurable disorder caused by mutations in the largest human gene: dystrophin. The combination of large-capacity vectors, such as human artificial chromosomes (HACs), with...

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PMID: 29242210
DOI: 10.15252/emmm.201607284
PMCID: PMC5801502

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