公开(公告)号：US5635385A

公开(公告)日：1997-06-03

申请号：US122795

申请日：1993-09-15

申请人： Lance H. Leopold ,  Scott K. Shore ,  Moole V. R. Reddy ,  E. Premkumar Reddy

发明人： Lance H. Leopold ,  Scott K. Shore ,  Moole V. R. Reddy ,  E. Premkumar Reddy

IPC分类号： A61K38/00 ,  A61K47/48 ,  A61K48/00 ,  C12N15/113 ,  C12N5/08 ,  C07H21/02 ,  C12N5/16 ,  C12N15/85

CPC分类号： C12N15/1135 ,  A61K47/48276 ,  A61K47/48315 ,  A61K48/00 ,  A61K38/00 ,  C12N2310/111 ,  C12N2310/12 ,  C12N2310/351 ,  C12N2310/3513 ,  C12N2310/3515

摘要： A novel multi-unit ribozyme is provided which is capable of selectively cleaving the mRNA transcript of a hybrid oncogene resulting from a chromosomal translocation. Specialized delivery vehicles such as liposomes, and growth factor conjugates for cellular uptake by receptor-mediated endocytosis, are also described. The multi-unit ribozyme is used for the treatment of neoplasms characterized by expression of the oncogene. In one embodiment, a multi-unit ribozyme is provided which cleaves the mRNA transcript of Philadelphia chromosome-positive cells, thereby blocking production of the tumorigenic p210.sup.bcr-abl fusion protein.

摘要翻译： 提供了一种新颖的多单位核酶，其能够选择性地切割由染色体易位引起的杂合癌基因的mRNA转录物。 还描述了专用递送载体，例如脂质体和用于通过受体介导的内吞作用进行细胞摄取的生长因子缀合物。 多单元核酶用于治疗以癌基因表达为特征的肿瘤。 在一个实施方案中，提供多单位核酶，其切割费城染色体阳性细胞的mRNA转录物，从而阻断致瘤性p210bcr-abl融合蛋白的产生。