Ribozymes in the age of molecular therapeutics.
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.
Bagheri
Sepideh
S
Auerback Melanoma Research Laboratory, Cutaneous Oncology Program, UCSF Comprehensive Cancer Center and Department of Dermatology, San Francisco, CA 94115, USA.
Kashani-Sabet
Mohammed
M
D000818:Animals; D030342:Genetic Diseases, Inborn / Q000235:genetics / Q000628:therapy*; D015316:Genetic Therapy / Q000639:trends; D006801:Humans; D007438:Introns / Q000235:genetics; D009369:Neoplasms / Q000235:genetics / Q000628:therapy*; D009690:Nucleic Acid Conformation; D012326:RNA Splicing / Q000235:genetics; D016337:RNA, Catalytic / Q000737:chemistry / Q000235:genetics* / Q000627:therapeutic use*; D012333:RNA, Messenger / Q000235:genetics / Q000378:metabolism; D043262:Ribonuclease P / Q000235:genetics / Q000378:metabolism; D014777:Virus Diseases / Q000235:genetics / Q000628:therapy*
D016337:RNA, Catalytic; D012333:RNA, Messenger; D043262:Ribonuclease P