Article

Advanced human iPSC-based modelling of <i>LMNA</i> -related congenital muscular dystrophy enables development of targeted genetic therapies for muscle laminopathies

2025-06-23

Abstract excerpt

<h4>ABSTRACT</h4> LMNA -related congenital muscular dystrophy (L-CMD) is amongst the most severe forms of laminopathies, which are diseases caused by pathogenic variants in the LMNA gene. LMNA encodes the proteins Lamin A and C, which assemble with Lamin B1 and B2 to form the nuclear lamina: a meshwork providing structural stability to the nucleus that also regulates chromatin organisation and gene expression....

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Literature Corpus work: c191c680-7de4-55d3-99dd-9a6d7227a595
DOI: 10.1101/2025.06.22.660928

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