Item Type: | Dataset |
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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 |
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