CELL ABLATION USING TRANS-SPLICING RIBOZYMES.

摘要

PCT No. PCT/US92/00277 Sec. 371 Date Jul. 14, 1993 Sec. 102(e) Date Dec. 23, 1993 PCT Filed Jan. 16, 1992 PCT Pub. No. WO92/13089 PCT Pub. Date Aug. 6, 1992The design of new ribozymes capable of self-catalyzed trans-splicing which are base a upon the catalytic core of a Group I intron are described. Using this design, it is possible to construct ribozymes capable of efficiently splicing a new 3' exon sequence into any chosen target RNA sequence in a highly precise manner. A method of cell ablation is also described that provides a toxic product to a host cell in vivo in a targetted, regulated manner utilizing novel trans-splicing ribozymes of the invention. Inactive pro-ribozyme forms are also described.