The potential application of ribozymes for the treatment of hematological disorders.
With the identification and increasing understanding of the genes involved in neoplastic transformation has come the realization that abrogation of these genes' products may lead to cell death or a return to normalcy. The use of ribozymes and their nucleic acid cousins, antisense oligodeoxynucleotides (ODNs), are two such ways of perturbing the disease-related gene expression. This review will look at the development and application of ribozymes to abrogate gene expression, with particular relevance to hematological settings. Some examples of antisense ODNs will also be mentioned where appropriate.
James
H A
HA
School of Biological Sciences, University of East Anglia, Norwich, Norfolk, UK. h.a.james@uea.ac.uk
D019008:Drug Resistance, Neoplasm / Q000235:genetics; D005786:Gene Expression Regulation / Q000187:drug effects*; D015316:Genetic Therapy; D006402:Hematologic Diseases / Q000235:genetics / Q000628:therapy*; D019337:Hematologic Neoplasms / Q000235:genetics / Q000628:therapy; D006801:Humans; D007154:Immune System Diseases / Q000628:therapy; D009690:Nucleic Acid Conformation; D016376:Oligonucleotides, Antisense / Q000493:pharmacokinetics / Q000494:pharmacology / Q000627:therapeutic use; D015514:Oncogene Proteins, Fusion / Q000235:genetics; D009857:Oncogenes / Q000235:genetics; D016337:RNA, Catalytic / Q000737:chemistry / Q000493:pharmacokinetics / Q000494:pharmacology / Q000627:therapeutic use*; D014777:Virus Diseases / Q000628:therapy; D014779:Virus Replication / Q000187:drug effects
D016376:Oligonucleotides, Antisense; D015514:Oncogene Proteins, Fusion; D016337:RNA, Catalytic