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Updated: Jun 27, 2025

Efficient PAM-Less Base Editing for Zebrafish Modeling of Human Genetic Disease with zSpRY-ABE8e
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Published on: February 17, 2023

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背景基础编辑用于IVSI-110β-thalassemia的拼接校正

Basma Naiisseh1, Panayiota L Papasavva1, Nikoletta Y Papaioannou1

  • 1Molecular Genetics of Thalassemia Department, The Cyprus Institute of Neurology & Genetics, 6 Iroon Avenue, Agios Dometios, Nicosia 2371, Cyprus.

Molecular therapy. Nucleic acids
|May 6, 2024
PubMed
概括

基地编辑器精确地纠正患者细胞中的HBB拼接突变,为β-thalassemia提供潜在的治愈疗法. 这种基因编辑方法在RNA,蛋白质和细胞分化水平上显示出高效率.

关键词:
这就是CRISPR/Cas9的作用.MT:编辑RNA/DNA的方法基础编辑 基础编辑基因治疗的基因疗法造血干细胞和原始细胞的产生.血红蛋白病变 - 血红蛋白病变几乎没有PAM的PAM.核作用的核作用.在β-thalassemia中,

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