RNAi in combination with a ribozyme and TAR decoy for treatment of HIV infection in hematopoietic cell gene therapy

Mingjie Li, Haitang Li, John J. Rossi

Research output: Chapter in Book/Report/Conference proceedingConference contributionpeer-review

49 Scopus citations


Combinatorial therapies for the treatment of HIV infection have changed the course of the AIDS epidemic in developed nations where the antiviral drug combinations are readily available. Despite this progress, there are many problems associated with chemotherapy for AIDS including toxicities and emergence of viral mutants resistant to the drugs. Our goal has been the development of a hematopoietic gene therapy treatment for HIV infection. Like chemotherapy, gene therapy for treatment of HIV infection should be used combinatorially. We have thus combined three different inhibitory genes for treatment of HIV infection into a single lentiviral vector backbone. The inhibitory agents engage RNAi via a short hairpin RNA targeting HIV tat/rev mRNAs, a nucleolar localizing decoy that binds and sequesters the HIV Tat protein, and a ribozyme that cleaves and downregulates the CCR5 chemokine receptor used by HIV for cellular entry. This triple combination has proven to be highly effective for inhibiting HIV replication in primary hematopoietic cells, and is currently on track for human clinical application.

Original languageEnglish
Title of host publicationOligonucleotide Therapeutics
Subtitle of host publicationFirst Annual Meeting of the Oligonucleotide Therapeutics Society
PublisherBlackwell Publishing Inc.
Number of pages8
ISBN (Print)1573315877, 9781573315876
StatePublished - Oct 2006

Publication series

NameAnnals of the New York Academy of Sciences
ISSN (Print)0077-8923
ISSN (Electronic)1749-6632


  • AIDS
  • HIV
  • Oligonucleotides
  • RNAi
  • Ribozyme
  • TAR decoy
  • shRNA


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