首页> 外国专利> RAAV VECTOR COMPOSITIONS, METHODS FOR TARGETING VASCULAR ENDOTHELIAL CELLS AND USE IN TREATMENT OF TYPE I DIABETES

RAAV VECTOR COMPOSITIONS, METHODS FOR TARGETING VASCULAR ENDOTHELIAL CELLS AND USE IN TREATMENT OF TYPE I DIABETES

机译:RAAV矢量成分,靶向血管内皮细胞的方法及其在I型糖尿病治疗中的用途

摘要

Disclosed are capsid-modified rAAV expression vectors, as well as infectious virions, compositions, and pharmaceutical formulations that include them. Also disclosed are methods of preparing and using novel capsid-protein-mutated rAAV vector constructs in a variety of diagnostic and therapeutic applications including, inter alia, as delivery agents for diagnosis, treatment, or amelioration of one or more diseases, disorders, or dysfunctions of the mammalian vascular system, and complications from Type I diabetes. Also disclosed are methods for systemic and tissue-localized delivery of therapeutic rAAV-based gene expression cassettes to vascular endothelial cells, tissues, and organs, as well as use of the disclosed compositions in the manufacture of medicaments for a variety of in vitro and/or in vivo applications including the treatment of vasculitis, and complications arising from Type I diabetes, such as macular edema, nephropathy, diabetic retinopathy, and the like.
机译:公开了衣壳修饰的rAAV表达载体,以及包括它们的感染性病毒体,组合物和药物制剂。还公开了在各种诊断和治疗应用中制备和使用新颖的衣壳蛋白突变的rAAV载体构建体的方法,所述应用尤其包括作为用于诊断,治疗或改善一种或多种疾病,病症或功能障碍的递送剂。血管系统疾病以及I型糖尿病引起的并发症。还公开了将治疗性基于rAAV的基因表达盒全身和组织定位递送至血管内皮细胞,组织和器官的方法,以及所公开的组合物在制备用于多种体外和/或药物的药物中的用途。或包括血管炎和I型糖尿病引起的并发症(如黄斑水肿,肾病,糖尿病性视网膜病等)在内的体内应用。

著录项

相似文献

  • 专利
  • 外文文献
  • 中文文献
获取专利

客服邮箱:kefu@zhangqiaokeyan.com

京公网安备:11010802029741号 ICP备案号:京ICP备15016152号-6 六维联合信息科技 (北京) 有限公司©版权所有
  • 客服微信

  • 服务号