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首页> 外国专利> PROCESS OF GENE-EDITING OF CELLS ISOLATED FROM A SUBJECT SUFFERING FROM A METABOLIC DISEASE AFFECTING THE ERYTHROID LINEAGE, CELLS OBTAINED BY SAID PROCESS AND USES THEREOF

PROCESS OF GENE-EDITING OF CELLS ISOLATED FROM A SUBJECT SUFFERING FROM A METABOLIC DISEASE AFFECTING THE ERYTHROID LINEAGE, CELLS OBTAINED BY SAID PROCESS AND USES THEREOF

机译：患有患红细胞系的代谢性疾病的受试者分离的细胞的基因编辑过程，上述方法获得的细胞及其用途

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The present invention relates to the medical field, in particular to gene editing as a therapeutic approach for the treatment of metabolic diseases affecting the erythroid lineage in a mammalian subject. In invention particular embodiment it refers to the combination of cell reprograming and gene editing for PKD correction as a first example of the potential application of these advanced technologies to metabolic diseases affecting the erythroid lineage. In this sense, PKD patient-specific iPSCs were efficiently generated from PB-MNCs (peripheral blood mononuclear cells) by a SeV non-integrative system and efficiently use to treat pyruvate kinase deficiency. The gene editing strategy for PKLR gene correction was also successfully applied directly to hematopoietic progenitors.
机译：本发明涉及医学领域，尤其涉及基因编辑，作为治疗影响哺乳动物受试者中红系谱系的代谢疾病的治疗方法。在本发明的特定实施方案中，它是指用于PKD校正的细胞重编程和基因编辑的组合，作为这些先进技术在影响红系谱系的代谢疾病中潜在应用的第一个例子。从这个意义上讲，通过SeV非整合系统从PB-MNC（外周血单核细胞）有效地产生了PKD患者特异性iPSC，并有效地用于治疗丙酮酸激酶缺乏症。 PKLR基因校正的基因编辑策略也已成功地直接应用于造血祖细胞。

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