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Ribozymes in the Age of Molecular Therapeutics

[ Vol. 4 , Issue. 5 ]

Author(s):

Sepideh Bagheri and Mohammed Kashani-Sabet   Pages 489 - 506 ( 18 )

Abstract:


Ribozymes are RNA molecules capable of sequence-specific cleavage of other RNA molecules. Since the discovery of the first group I intron ribozyme in 1982, new classes of ribozymes, each with their own unique reaction, target site specifications, and potential applications, have been identified. These include hammerhead, hairpin, hepatitis delta, varkud satellite, groups I and II intron, and RNase P ribozymes, as well as the ribosome and spliceosome. Meanwhile, ribozyme engineering has enabled the in vitro selection of synthetic ribozymes with unique properties. This, along with advances in ribozyme delivery methods and expression systems, has led to an explosion in the potential therapeutic applications of ribozymes, whether for anti-cancer or anti-viral therapy, or for gene repair.

Keywords:

ribozymes, gene therapy, gene repair, rna catalysis, cancer, anti-viral therapy, molecular, therapeutics, repair of heritable diseases

Affiliation:

Melanoma Center,UCSF Comprehensive Cancer Center, 1600 Divisadero Street, 2ndFloor, Box 1706, San Francisco, CA 94115, USA



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