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ASSAY FOR SAFETY ASSESSMENT OF THERAPEUTIC GENETIC MANIPULATIONS, GENE THERAPY VECTORS AND COMPOUNDS
ASSAY FOR SAFETY ASSESSMENT OF THERAPEUTIC GENETIC MANIPULATIONS, GENE THERAPY VECTORS AND COMPOUNDS
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机译:治疗性遗传操作,基因治疗载体和化合物的安全性评估
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摘要
The invention is directed to methods of assessing the safety of therapeutic compounds and therapeutic genetic manipulations, including integrating gene therapy vectors and genome editing. In particular, the invention provides a method, wherein the oncogenic potential of therapeutic compounds and therapeutic genetic manipulations, including integrating gene therapy vectors and genome editing, is determined by determining the percentage of differentiation blocked hematopoietic progenitor cells.
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