Document 0802 DOCN M94B0802 TI Ribozyme targeting of HIV-1 LTR. DT 9412 AU Ventura M; Wang P; Franck N; Saragosti S; ICGM, INSERM U 363, Universite Paris V, Hospital Cochin, Paris,; France. SO Biochem Biophys Res Commun. 1994 Sep 15;203(2):889-98. Unique Identifier : AIDSLINE MED/94380073 AB The 5'-TAR region of HIV-1 mRNA is highly conserved amongst different HIV-1 isolates. We thus investigated the potential for in vivo targeting of the TAR RNA element by a hammerhead ribozyme. The use of the CAT reporter gene linked to the HIV1-LTR, in transient assays, reveals that a hammerhead ribozyme directed towards the first GUC of HIV-1 mRNA can efficiently inhibit CAT protein expression. We show that this inhibition is sequence-specific and probably due to a cleavage activity rather than an antisense effect. We show also that a hammerhead ribozyme that is inactive in vitro is capable of inhibiting CAT protein expression in a cellular environment. These results suggest that the targeting of the HIV-1 LTR by a hammerhead ribozyme constitutes a viable approach for anti-HIV therapy. DE Base Sequence Cell Line Chloramphenicol Acetyltransferase/GENETICS Gene Expression Gene Products, tat/GENETICS/PHARMACOLOGY Genes, Reporter HIV Long Terminal Repeat/*GENETICS HIV-1/*GENETICS Molecular Sequence Data Plasmids RNA, Catalytic/ANTAGONISTS & INHIB/CHEMISTRY/*METABOLISM RNA, Messenger/METABOLISM Transfection JOURNAL ARTICLE SOURCE: National Library of Medicine. NOTICE: This material may be protected by Copyright Law (Title 17, U.S.Code).