<i>LMNA</i> Co-Regulated Gene Expression as a Suitable Readout after Precise Gene Correction

<i>LMNA</i> Co-Regulated Gene Expression as a Suitable Readout after Precise Gene Correction

<i>LMNA</i>-related muscular dystrophy is an autosomal-dominant progressive disorder caused by mutations in <i>LMNA</i>. <i>LMNA</i> missense mutations are becoming correctable with CRISPR/Cas9-derived tools. Evaluating the functional recovery of <i>LMNA<...

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Bibliographic Details
Main Authors: Haicui Wang, Anne Krause, Helena Escobar, Stefanie Müthel, Eric Metzler, Simone Spuler
Format: Article
Language:English
Published: MDPI AG 2022-12-01
Series:International Journal of Molecular Sciences
Subjects:
laminopathy
muscular dystrophy
<i>LMNA</i> co-regulated genes
near-PAMless cytosine base editor
serum-induced differentiation (SID)
patient-derived induced pluripotent stem cells (iPSCs)
Online Access:https://www.mdpi.com/1422-0067/23/24/15525

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https://www.mdpi.com/1422-0067/23/24/15525

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