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TARGETING THE HUMAN CCR5 LOCUS AS A SAFE HARBOR FOR THE EXPRESSION OF THERAPEUTIC PROTEINS
TARGETING THE HUMAN CCR5 LOCUS AS A SAFE HARBOR FOR THE EXPRESSION OF THERAPEUTIC PROTEINS
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机译:针对人CCR5基因座作为治疗蛋白表达的安全港
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摘要
The present disclosure provides methods and compositions for treating lysosomal storage disorders in subjects, comprising genetically modifying cells from the subjects ex vivo by integrating therapeutic transgenes into the CCR5 locus.
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