Gene Therapy Strategies for HIV/AIDS

A special issue of Biomedicines (ISSN 2227-9059).

Deadline for manuscript submissions: closed (30 January 2016) | Viewed by 14040

Special Issue Editor


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Guest Editor
Department of Pharmaceutical Sciences, Washington State University, Pullman, WA 99164, USA
Interests: hematopoietic stem cell gene therapy; foamy retroviral vectors; vector-mediated genotoxicity; retroviral mutagenesis screens for cancer drivers

Special Issue Information

Dear Colleagues,

This Special Issue “Gene Therapy Strategies for HIV/AIDS” will focus on novel approaches to treat HIV/AIDS using gene transfer.

The cure of the “Berlin patient” by transplantation of allogeneic hematopoietic stem cells has provided a proof of principle that HIV/AIDS can be treated using genetically resistant stem cells. This has led to preclinical and clinical studies exploring numerous approaches to create genetically modified hematopoietic stem cells and T cells that resist HIV infection. In addition, means to deliver transgenes encoding broadly neutralizing antibodies or antibody-fusions to muscle cells have shown great promise.

This issue will focus on novel approaches to HIV gene therapy, vector efficacy and safety, preclinical models and immunogenicity, and translation to the clinic.

Dr. Grant Trobridge
Guest Editor

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Keywords

  • HIV gene therapy
  • viral vector
  • stem cell gene therapy
  • gene targeting
  • preclinical models

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Published Papers (2 papers)

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Review

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Review
Prospects for Foamy Viral Vector Anti-HIV Gene Therapy
by Arun K. Nalla and Grant D. Trobridge
Biomedicines 2016, 4(2), 8; https://doi.org/10.3390/biomedicines4020008 - 29 Mar 2016
Cited by 3 | Viewed by 7177
Abstract
Stem cell gene therapy approaches for Human Immunodeficiency Virus (HIV) infection have been explored in clinical trials and several anti-HIV genes delivered by retroviral vectors were shown to block HIV replication. However, gammaretroviral and lentiviral based retroviral vectors have limitations for delivery of [...] Read more.
Stem cell gene therapy approaches for Human Immunodeficiency Virus (HIV) infection have been explored in clinical trials and several anti-HIV genes delivered by retroviral vectors were shown to block HIV replication. However, gammaretroviral and lentiviral based retroviral vectors have limitations for delivery of anti-HIV genes into hematopoietic stem cells (HSC). Foamy virus vectors have several advantages including efficient delivery of transgenes into HSC in large animal models, and a potentially safer integration profile. This review focuses on novel anti-HIV transgenes and the potential of foamy virus vectors for HSC gene therapy of HIV. Full article
(This article belongs to the Special Issue Gene Therapy Strategies for HIV/AIDS)
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1196 KiB  
Review
Insulators to Improve the Safety of Retroviral Vectors for HIV Gene Therapy
by Diana L. Browning and Grant D. Trobridge
Biomedicines 2016, 4(1), 4; https://doi.org/10.3390/biomedicines4010004 - 2 Feb 2016
Cited by 9 | Viewed by 6283
Abstract
Retroviral vector gene therapy is a promising approach to treating HIV-1. However, integrated vectors are mutagens with the potential to dysregulate nearby genes and cause severe adverse side effects. Leukemia has already been a documented severe adverse event in gene therapy clinical trials [...] Read more.
Retroviral vector gene therapy is a promising approach to treating HIV-1. However, integrated vectors are mutagens with the potential to dysregulate nearby genes and cause severe adverse side effects. Leukemia has already been a documented severe adverse event in gene therapy clinical trials for the treatment of primary immunodeficiencies. These side effects will need to be reduced or avoided if retroviral vectors are to be used clinically for HIV-1 treatment. The addition of chromatin insulators to retroviral vectors is a potential strategy for reducing adverse side effects. Insulators have already been effectively used in retroviral vectors to reduce genotoxicity in pre-clinical studies. Here, we will review how insulators function, genotoxicity in gene therapy clinical trials, the design of insulated retroviral vectors, promising results from insulated retroviral vector studies, and considerations for the development of insulated retroviral treatment vectors for HIV-1 gene therapy. Full article
(This article belongs to the Special Issue Gene Therapy Strategies for HIV/AIDS)
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