Development of Hematopoietic Stem Cell Based Gene Therapy for HIV-1 Infection: Considerations for Proof of Concept Studies and Translation to Standard Medical Practice

David L Digiusto, Rodica Stan, A K Sri Krishnan, Haitang Li, John J. Rossi, John A Zaia
Published 2013 in Viruses

Abstract

Over the past 15 years we have been investigating an alternative approach to treating HIV-1/AIDS, based on the creation of a disease-resistant immune system through transplantation of autologous, gene-modified (HIV-1-resistant) hematopoietic stem and progenitor cells (GM-HSPC). We propose that the expression of selected RNA-based HIV-1 inhibitors in the CD4… (More)

DOI: 10.3390/v5112898

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Long-term inhibition of HIV-1 infection in primary hematopoietic cells by lentiviral vector delivery of a triple combination of anti-HIV shRNA, anti-CCR5 ribozyme, and a nucleolar-localizing TAR decoy.

Ming-Jie Li, James Kim, +5 authors John J Rossi
Molecular therapy : the journal of the American…
2005

Development of Hematopoietic Stem Cell Based Gene Therapy for HIV-1 Infection: Considerations for Proof of Concept Studies and Translation to Standard Medical Practice

Abstract

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Citation Velocity: 16

Cited By

Polymers in the Delivery of siRNA for the Treatment of Virus Infections.

RNA Interference Therapies for an HIV-1 Functional Cure

The therapeutic landscape of HIV-1 via genome editing

Development of Lentiviral Vectors Simultaneously Expressing Multiple siRNAs Against CCR5, vif and tat/rev Genes for an HIV-1 Gene Therapy Approach

Preclinical development and qualification of ZFN-mediated CCR5 disruption in human hematopoietic stem/progenitor cells

Prospects for Foamy Viral Vector Anti-HIV Gene Therapy

Stem Cells Applications in Regenerative Medicine and Disease Therapeutics

Participation in HIV cure-related research: a scoping review of the proxy literature and implications for future research

Progress toward curing HIV infection with hematopoietic cell transplantation

RNA interference approaches for treatment of HIV-1 infection

References

Gene therapy for adenosine deaminase-deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans.

Long-term inhibition of HIV-1 infection in primary hematopoietic cells by lentiviral vector delivery of a triple combination of anti-HIV shRNA, anti-CCR5 ribozyme, and a nucleolar-localizing TAR decoy.

CD133+ hematopoietic progenitor cells harbor HIV genomes in a subset of optimally treated people with long-term viral suppression.

Challenges in Detecting HIV Persistence during Potentially Curative Interventions: A Study of the Berlin Patient

Economic savings versus health losses: the cost-effectiveness of generic antiretroviral therapy in the United States.

HIV-1 infection of hematopoietic progenitor cells in vivo in humanized mice.

Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy.

Lentiviral hematopoietic stem cell gene therapy in patients with wiskott-Aldrich syndrome.

Long-term reduction in peripheral blood HIV type 1 reservoirs following reduced-intensity conditioning allogeneic stem cell transplantation.

Positive selection of mC46-expressing CD4+ T cells and maintenance of virus specific immunity in a primate AIDS model.

Slides referencing similar topics

Development of Hematopoietic Stem Cell Based Gene Therapy for HIV-1 Infection: Considerations for Proof of Concept Studies and Translation to Standard Medical Practice

Abstract

Topics

4 Figures and Tables

Statistics

Citation Velocity: 16

Cited By

Polymers in the Delivery of siRNA for the Treatment of Virus Infections.

RNA Interference Therapies for an HIV-1 Functional Cure

The therapeutic landscape of HIV-1 via genome editing

Development of Lentiviral Vectors Simultaneously Expressing Multiple siRNAs Against CCR5, vif and tat/rev Genes for an HIV-1 Gene Therapy Approach

Preclinical development and qualification of ZFN-mediated CCR5 disruption in human hematopoietic stem/progenitor cells

Prospects for Foamy Viral Vector Anti-HIV Gene Therapy

Stem Cells Applications in Regenerative Medicine and Disease Therapeutics

Participation in HIV cure-related research: a scoping review of the proxy literature and implications for future research

Progress toward curing HIV infection with hematopoietic cell transplantation

RNA interference approaches for treatment of HIV-1 infection

References

Gene therapy for adenosine deaminase-deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans.

Long-term inhibition of HIV-1 infection in primary hematopoietic cells by lentiviral vector delivery of a triple combination of anti-HIV shRNA, anti-CCR5 ribozyme, and a nucleolar-localizing TAR decoy.

CD133+ hematopoietic progenitor cells harbor HIV genomes in a subset of optimally treated people with long-term viral suppression.

Challenges in Detecting HIV Persistence during Potentially Curative Interventions: A Study of the Berlin Patient

Economic savings versus health losses: the cost-effectiveness of generic antiretroviral therapy in the United States.

HIV-1 infection of hematopoietic progenitor cells in vivo in humanized mice.

Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy.

Lentiviral hematopoietic stem cell gene therapy in patients with wiskott-Aldrich syndrome.

Long-term reduction in peripheral blood HIV type 1 reservoirs following reduced-intensity conditioning allogeneic stem cell transplantation.

Positive selection of mC46-expressing CD4+ T cells and maintenance of virus specific immunity in a primate AIDS model.

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