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1.发明授权公开(公告)号：US11268158B2
公开(公告)日：2022-03-08
申请号：US15136298
申请日：2016-04-22
Applicant: ST. JUDE CHILDREN'S RESEARCH HOSPITAL, INC.
Inventor： Brian P. Sorrentino , Sheng Zhou
IPC: C12Q1/70
Abstract: 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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2.发明申请ASSAY FOR SAFETY ASSESSMENT OF THERAPEUTIC GENETIC MANIPULATIONS, GENE THERAPY VECTORS AND COMPOUNDS 审中-公开治疗基因治疗安全评估方法，基因治疗药物和化合物公开(公告)号：US20160312304A1
公开(公告)日：2016-10-27
申请号：US15136298
申请日：2016-04-22
Applicant: ST. JUDE CHILDREN'S RESEARCH HOSPITAL, INC.
Inventor： Brian P. SORRENTINO , Sheng Zhou
CPC classification number: C12Q1/702
Abstract: 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.
Abstract translation: 本发明涉及评估治疗化合物的安全性和治疗性遗传操作的方法，包括整合基因治疗载体和基因组编辑。 特别地，本发明提供了一种方法，其中通过确定分化阻断的造血祖细胞的百分比来确定治疗化合物的致癌潜力和治疗性遗传操作，包括整合基因治疗载体和基因组编辑。
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