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CRISPR/Cas9 Genome Editing in LGMD2A/R1 Patient-Derived Induced Pluripotent Stem and Skeletal Muscle Progenitor Cells

Large numbers of Calpain 3 (CAPN3) mutations cause recessive forms of limb-girdle muscular dystrophy (LGMD2A/LGMDR1) with selective atrophy of the proximal limb muscles. We have generated induced pluripotent stem cells (iPSC) from a patient with two mutations in exon 3 and exon 4 at the calpain 3 lo...

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Detalles Bibliográficos
Autores principales:	Mavrommatis, Lampros, Zaben, Abdul, Kindler, Urs, Kienitz, Marie-Cécile, Dietz, Julienne, Jeong, Hyun-Woo, Böhme, Pierre, Brand-Saberi, Beate, Vorgerd, Matthias, Zaehres, Holm
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Hindawi 2023
Materias:	Research Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10653971/ https://www.ncbi.nlm.nih.gov/pubmed/38020204 http://dx.doi.org/10.1155/2023/9246825

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