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Updated: Jul 26, 2025

Efficient PAM-Less Base Editing for Zebrafish Modeling of Human Genetic Disease with zSpRY-ABE8e
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基础编辑和主要编辑:对于罕见和常见疾病的潜在治疗选择

Lauren C Testa1, Kiran Musunuru2

  • 1Division of Cardiovascular Medicine, Department of Genetics, Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.

BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy
|June 14, 2023
PubMed
概括

基因编辑 (BE) 和主要编辑 (PE) 为遗传疾病提供了精确,安全的基因组编辑. 这些CRISPR-Cas9技术在没有双链断裂的情况下纠正致病变体,从而推进潜在的罕见疾病治疗方法.

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