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Prime Editing Corrects RBM20-Mediated Dilated Cardiomyopathy in Human Cardiomyocytes

Item Type:Dataset
Title:Prime Editing Corrects RBM20-Mediated Dilated Cardiomyopathy in Human Cardiomyocytes
Abstract:The goal of the study is to establish Prime Editing (PE) in differentiated, non-dividing human cardiomyocytes to precisely repair patient-derived DCM mutations to wildtype. We focus on three pathogenic DCM-causing mutations within the human LMNA and RBM20 genes. Our results support further pre-clinical development of PE-based therapies, particularly for RBM20-P633L-associated DCM.
Source:BioProject
Publisher:National Library of Medicine
Date:2 July 2025
External Fulltext:View full text on external repository or document server
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