Article
LMNA Co-Regulated Gene Expression as a Suitable Readout after Precise Gene Correction.
International journal of molecular sciences - 8 Dec 2022
Wang Haicui, Krause Anne, Escobar Helena, Müthel Stefanie, Metzler Eric, Spuler Simone
Abstract excerpt
LMNA-related muscular dystrophy is an autosomal-dominant progressive disorder caused by mutations in LMNA. LMNA missense mutations are becoming correctable with CRISPR/Cas9-derived tools. Evaluating the functional recovery of LMNA after gene editing bears challenges as there is no reported direct loss of function of lamin A/C proteins in patient-derived cells. The proteins encoded by LMNA are lamins A/C,...
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