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TARGETED INTEGRATION SYSTEMS AND METHODS FOR THE TREATMENT OF HEMOGLOBINOPATHIES
TARGETED INTEGRATION SYSTEMS AND METHODS FOR THE TREATMENT OF HEMOGLOBINOPATHIES
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机译:针对性血红蛋白病的靶向整合系统和方法
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摘要
Genome editing systems, guide RNAs, DNA donor templates, and CRISPR-mediated methods are provided for altering a β-globin gene to alter a genotype, e.g., by correcting, or partially correcting, a genotype associated with thalassemia or sickle cell disease.
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