Design and testing of ribozymes for cancer gene therapy.
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.
Norris
J S
JS
Department of Microbiology and Immunology, Medical University of South Carolina, Charleston 29425, USA.
Hoel
B
B
Voeks
D
D
Maggouta
F
F
Dahm
M
M
Pan
W
W
Clawson
G
G
D000818:Animals; D001483:Base Sequence; D015316:Genetic Therapy / Q000379:methods; D006801:Humans; D008297:Male; D008969:Molecular Sequence Data; D009369:Neoplasms / Q000628:therapy; D011471:Prostatic Neoplasms / Q000628:therapy*; D012318:RNA Polymerase I / Q000235:genetics* / Q000378:metabolism; D016337:RNA, Catalytic / Q000235:genetics* / Q000378:metabolism
D016337:RNA, Catalytic; D012318:RNA Polymerase I