Design and testing of ribozymes for cancer gene therapy

Adv Exp Med Biol. 2000:465:293-301. doi: 10.1007/0-306-46817-4_25.

Abstract

This chapter describes procedural aspects for development of ribozymes in general, and specifically, that cleave mRNA to an essential cellular gene, the AC40 subunit of RNA pol I. Ribozyme design includes functional selection of binding sites followed by computer modeling. These ribozymes are being used in vectors that target expression to the prostate via tissue specific promoters (Voeks, Norris, and Clawson, 1998) and have demonstrated efficacy.

Publication types

  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.
  • Review

MeSH terms

  • Animals
  • Base Sequence
  • Genetic Therapy* / methods
  • Humans
  • Male
  • Molecular Sequence Data
  • Neoplasms / therapy
  • Prostatic Neoplasms / therapy*
  • RNA Polymerase I / genetics*
  • RNA Polymerase I / metabolism
  • RNA, Catalytic / genetics*
  • RNA, Catalytic / metabolism

Substances

  • RNA, Catalytic
  • RNA Polymerase I