Prime Editing Corrects RBM20-Mediated Dilated Cardiomyopathy in Human Cardiomyocytes
收藏NIAID Data Ecosystem2026-05-02 收录
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https://www.ncbi.nlm.nih.gov/sra/SRP596918
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资源简介：
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
创建时间：
2025-07-04
