Cell ablation using trans-splicing ribozymes
AssigneesThe General Hospital Corporation, President and Fellows of Harvard College
InventorsJames Haseloff, Andrea Brand, Norbert Perrimon, Howard M. Goodman
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Abstract
The 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.
Claims25
A polynucleotide molecule, said molecule encoding a trans-splicing Group I ribozyme, the sequence of said ribozyme being a fusion RNA, the sequence of said fusion RNA comprising:
- (1) a first RNA sequence which hybridizes to a target RNA that encodes a transcription activator protein, and
- (2) a second RNA sequence being a sequence to be trans-spliced into said target RNA;
- wherein the nucleotide sequence encoding said ribozyme is operably linked to a transcription regulatory element which is specifically recognized by said transcription activator protein such that association of said transcription activator protein with said transcription regulatory element results in activation of transcription of said nucleotide sequence encoding said ribozyme.
Dependencies are inferred from the claim text.