首页> 外国专利> CELL FOR GENE THERAPY OF LCAT DEFICIENCY, AND REPLICATION-DEFICIENT RETROVIRUS VECTOR AND PLASMID FOR USE IN PRODUCTION OF THE CELL

CELL FOR GENE THERAPY OF LCAT DEFICIENCY, AND REPLICATION-DEFICIENT RETROVIRUS VECTOR AND PLASMID FOR USE IN PRODUCTION OF THE CELL

机译:用于LCAT缺陷基因治疗的细胞,以及复制缺陷型逆转录病毒和质粒用于细胞生产

摘要

PPROBLEM TO BE SOLVED: To provide a replication-deficient retrovirus vector carrying an LCAT-encoding nucleotide, which enables the LCAT-encoding nucleotide as an exogenous gene to be expressed in a target cell with efficiency, and which can be used for the production of cells for gene therapy to be administered to a patient, and a plasmid which can be used for the production of the retrovirus vector. PSOLUTION: Provided is the plasmid for the production of the replication-deficient retrovirus vector, having an LCAT-encoding nucleotide sequence which is inserted into between the 5'-LTR sequence and the 3'-LTR sequence originating from the retrovirus, as an exogenous gene, and a Kozak's consensus sequence and a packaging signal sequence for the retrovirus both are linked to the 5'-end of the LCAT-encoding nucleotide sequence. PCOPYRIGHT: (C)2009,JPO&INPIT
机译:

要解决的问题:提供带有LCAT编码核苷酸的复制缺陷型逆转录病毒载体,该载体可使作为外源基因的LCAT编码核苷酸在靶细胞中高效表达,并可用于提供给患者的用于基因治疗的细胞的生产,以及可用于生产逆转录病毒载体的质粒。

解决方案:提供了用于生产复制缺陷型逆转录病毒载体的质粒,该质粒具有LCAT编码核苷酸序列,该核苷酸序列插入了逆转录病毒的5'-LTR序列和3'-LTR序列之间,作为外源基因,逆转录病毒的Kozak共有序列和包装信号序列均与LCAT编码核苷酸序列的5'末端相连。

版权:(C)2009,日本特许厅&INPIT

著录项

  • 公开/公告号JP2009201518A

    专利类型

  • 公开/公告日2009-09-10

    原文格式PDF

  • 申请/专利权人 CELLGENTECH INC;

    申请/专利号JP20090142610

  • 发明设计人 WAKI ATSUO;ASO MASAYOSHI;FUKUCHI JUNICHI;

    申请日2009-06-15

  • 分类号C12N15/09;C12N5/10;A61K48;A61K35/12;C12N7/06;

  • 国家 JP

  • 入库时间 2022-08-21 19:44:46

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