A systematic review of adeno-associated virus gene therapy clinical trials for HIV – A potential solution for patients with haemophilia and HIV?

Sarina Levy-Mendelovich*, Christopher E. Walsh, Jonathan M. Schapiro, Shelly Soffer, Eyal Klang, Gili Kenet

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

Abstract

Introduction: In the past HIV infection was a common complication of haemophilia therapy. Gene therapy trials in Haemophilia patients using rAAV have shown promising results; Unfortunately, the majority of gene therapy trials studies have excluded HIV positive patients. We decided to systematically review the published clinical trials using rAAV for HIV prevention. Methods: A comprehensive literature search was performed to identify studies evaluating clinical trials using rAAV for HIV. The search was conducted using the MEDLINE/PubMed databases. Search keywords included ‘gene therapy’, ‘adeno-associated virus’, ‘HIV’ and ‘clinical trial’. Results: Three studies met our inclusion criteria. Two were phase 1 studies and one was a phase 2 study. One study examined an AAV coding for human monoclonal IgG1 antibody whereas the other two studies delivered a vector coding for viral protease and part of reverse transcriptase. All studies administered the vaccine intramuscularly and showed a response as well a good safety profile. Discussion: The concept of using a viral vector to prevent a viral infection is revolutionary. Due to the paucity of information regarding application of any gene therapy in HIV patients and the potential use of gene therapy in haemophilia patients with HIV in the future warrants attention.

Original languageEnglish
Pages (from-to)784-789
Number of pages6
JournalHaemophilia
Volume29
Issue number3
DOIs
StatePublished - May 2023

Keywords

  • HIV
  • adeno-associated virus
  • gene therapy
  • haemophilia
  • viral vector

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