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Dual CRISPR-Cas3 system for inducing multi-exon skipping in DMD patient-derived iPSCs

To restore dystrophin protein in various mutation patterns of Duchenne muscular dystrophy (DMD), the multi-exon skipping (MES) approach has been investigated. However, only limited techniques are available to induce a large deletion to cover the target exons spread over several hundred kilobases. He...

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Detalles Bibliográficos
Autores principales:	Kita, Yuto, Okuzaki, Yuya, Naoe, Youichi, Lee, Joseph, Bang, Uikyu, Okawa, Natsumi, Ichiki, Akane, Jonouchi, Tatsuya, Sakurai, Hidetoshi, Kojima, Yusuke, Hotta, Akitsu
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Elsevier 2023
Materias:	Report
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10545483/ https://www.ncbi.nlm.nih.gov/pubmed/37625413 http://dx.doi.org/10.1016/j.stemcr.2023.07.007

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