首页> 外国专利> COMPOSITIONS AND METHODS FOR ADENO-ASSOCIATED VIRUS MEDIATED GENE EXPRESSION IN MYOFIBROBLAST-LIKE CELLS

COMPOSITIONS AND METHODS FOR ADENO-ASSOCIATED VIRUS MEDIATED GENE EXPRESSION IN MYOFIBROBLAST-LIKE CELLS

机译:肌纤维母细胞样细胞中腺伴随病毒介导基因表达的组合物和方法

摘要

The present application discloses a recombinant AAV vector comprising a promoter that targets gene expression to myofibroblast-like lineage of cells in the heart. The present application, also discloses the preparation and use of AAV expression cassettes using a modified periostin promoter that successfully drives gene expression in cardiac myofibroblast-like cells. The present invention encompasses compositions and methods useful for treating myocardial infarction. Further comprising compositions and methods for preparing and using AAV vectors for targeting cells and inducing gene expression. The compositions and methods of the invention are useful for efficiently targeting cardiac myofibroblasts following a cardiac injury, disease, or disorder. Further comprising a kit for effecting alleviation of the various diseases or disorders recited herein.
机译:本申请公开了一种重组AAV载体,其包含将基因表达靶向心脏中成纤维细胞样谱系的启动子。本申请还公开了使用修饰的骨膜蛋白启动子的AAV表达盒的制备和使用,所述修饰的骨膜蛋白启动子成功地驱动心脏成纤维细胞样细胞中的基因表达。本发明包括可用于治疗心肌梗塞的组合物和方法。还包括用于制备和使用AAV载体靶向细胞并诱导基因表达的组合物和方法。本发明的组合物和方法可用于在心脏损伤,疾病或病症后有效地靶向心肌成纤维细胞。还包括用于实现本文所述的各种疾病或病症的缓解的试剂盒。

著录项

  • 公开/公告号WO2017151717A1

    专利类型

  • 公开/公告日2017-09-08

    原文格式PDF

  • 申请/专利权人 FRENCH BRENT A.;

    申请/专利号WO2017US20113

  • 发明设计人 FRENCH BRENT A.;

    申请日2017-03-01

  • 分类号A61K35/34;A61K48;A61P9/10;C12N15/63;

  • 国家 WO

  • 入库时间 2022-08-21 13:29:53

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