Multi-unit ribozyme inhibition of oncogene gene expression

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 p210bcr-abl fusion protein.