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Updated: Jan 11, 2026

CRISPR/Cas9 Editing of the C. elegans rbm-3.2 Gene using the dpy-10 Co-CRISPR Screening Marker and Assembled Ribonucleoprotein Complexes.
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CRISPR/Cas9 Editing of the C. elegans rbm-3.2 Gene using the dpy-10 Co-CRISPR Screening Marker and Assembled Ribonucleoprotein Complexes.

Published on: December 11, 2020

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主编辑纠正了导致人类心肌细胞RBM20-P633L突变的扩张性心肌病

Alexandra Roman1,2, Anja Zimmer1, Michael Gotthardt1,3,4

  • 1Max Delbrück Center for Molecular Medicine in the Helmholtz Association, Robert-Roessle-Strasse 10, 13125 Berlin, Germany.

Molecular therapy. Nucleic acids
|November 10, 2025
PubMed
概括

总编辑成功地纠正了人类心脏细胞中的扩展性心肌病变异. 这种基因编辑方法为非分裂细胞中遗传性心脏病提供了潜在的新疗法.

关键词:
克里斯普尔是什么意思?克里斯普尔是什么意思?LMNA LMNA 这是一个很大的问题.MT:RNA/DNA编辑 编辑在RBM20中使用RBM20基因编辑 基因编辑遗传性DCM是一种遗传性DCM.高心脏肌细胞 (hi-cardiomyocytes) 是一种高心脏肌细胞.主编辑主要编辑.

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