trans-splicing ribozymes and therapeutics

Ribozyme-mediated revision of RNA and DNA -- Long et al. 112 (3): 312 -- Journal of Clinical Investigation: "The therapeutic revision of RNA by ribozymes is mechanistically different from that of DNA. To revise RNA sequences, a Group I intron from Tetrahymena thermophila has been adapted for trans-splicing. Trans-splicing ribozymes splice therapeutic RNA sequences onto a target transcript in a process called ribozyme-mediated RNA repair. Considerable progress has been made in the development of trans-splicing ribozymes for therapeutic applications such as treating sickle cell disease and cancer." This is one of the relatively recent targeted genetic repair techniques.

"The spliceosome has also been shown to trans-splice RNA transcripts. Spliceosomal-mediated RNA trans-splicing is also called called SMaRT."

More detail pending:
Biochemistry Overview : Molecular Genetics Overview : SITE MAP : HOME