首页> 外国专利> EXPRESSION VECTORS WHICH INCLUDE THE HUMAN PKLR GENE PROMOTER AND USE THEREOF FOR PREPARING PHARMACEUTICAL COMPOSITIONS FOR SOMATIC GENETIC THERAPY WITH SPECIFIC EXPRESSION IN ERYTHROID CELLS

EXPRESSION VECTORS WHICH INCLUDE THE HUMAN PKLR GENE PROMOTER AND USE THEREOF FOR PREPARING PHARMACEUTICAL COMPOSITIONS FOR SOMATIC GENETIC THERAPY WITH SPECIFIC EXPRESSION IN ERYTHROID CELLS

机译:包含人类PKLR基因启动子的表达载体,并将其用于制备具有特定表达的红细胞中体细胞遗传疗法的药物组合物

摘要

Expression vectors which include the human PKLR promoter gene and use thereof for preparing pharmaceutical compositions for somatic genetic therapy with specific expression in erythroid cells. The present invention concerns the use of regulatory sequences of the promoter of the PKLR gene in the production of vectors, with the exception of vectors comprising adeno-associated viruses, for use in the preparation of pharmaceutical compositions intended for somatic genetic therapy in erythroid tissue.
机译:包括人PKLR启动子基因的表达载体及其在制备用于体细胞遗传治疗的药物组合物中的用途,所述药物组合物在类红细胞中特异性表达。本发明涉及PKLR基因启动子的调控序列在载体生产中的用途,除了包含腺相关病毒的载体以外,还用于制备用于类红细胞组织中的体细胞遗传疗法的药物组合物。

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