Lentivector Knockdown of CCR5 in Hematopoietic Stem and Progenitor Cells Confers Functional and Persistent HIV-1 Resistance in Humanized Mice

Myburgh, Renier; Ivic, Sandra; Pepper, Michael Sean; Gers-Huber, Gustavo; Li, Duo; Audigé, Annette; Rochat, Mary-Aude; Jaquet, Vincent; Regenass, Stephan; Manz, Markus G.; Salmon, Patrick; Krause, Karl‐Heinz; Speck, Roberto F.

doi:10.1128/jvi.00277-15

Cited by 32 publications

(27 citation statements)

References 54 publications

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“…the integration of short hairpin RNA (shRNA) or microRNA constructs, or the gene itself can be disrupted via gene-editing modalities (see below). In pre-clinical studies, this approach has led to a functional cure of HIV, [9] while clinical studies to assess their safety and efficacy are still underway.…”

Section: Researchmentioning

confidence: 99%

Transplantation of gene-modified haematopoietic stem cells: Application and clinical considerations

et al. 2019

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Section: Researchmentioning

confidence: 99%

Transplantation of gene-modified haematopoietic stem cells: Application and clinical considerations

et al. 2019

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“…In the field of the HIV research, LV have been extensively utilized for the delivery of anti-HIV genes that require stable expression for efficient HIV inhibition, such as CCR5 shRNA [28,79,99], LTR RNA [[28,32]], tat/rev shRNA, TRIM5α mutant, CCR5 ribozyme, TAR decoy [[22,79,86], C46 [[60]], or combinations of these genes. Anti-HIV genes transduced through HSPC have been stably expressed in vitro [[22,33,100]], in vivo animal models [[28,32,99]]. Although lentiviral vectors have been the first choice for anti-HIV HSPC gene delivery to CD34+ HSPC for a long term stable expression of anti-HIV genes.…”

Section: Anti Hiv Gene Delivery System For Anti-hiv Hspc Gene Therapymentioning

confidence: 99%

Stem cell-based therapies for HIV/AIDS

Pernet

Yadav

2016

Advanced Drug Delivery Reviews

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One of the current focuses in HIV/AIDS research is to develop a novel therapeutic strategy that can provide a life-long remission of HIV/AIDS without daily drug treatment and ultimately a cure for HIV/AIDS. Hematopoietic stem cell based anti-HIV gene therapy aims to reconstitute patient immune system by transplantation of genetically engineered hematopoietic stem cells with anti-HIV genes. Hematopoietic stem cells can self renew, proliferate and differentiate into mature immune cells. In theory, anti-HIV gene modified hematopoietic stem cells can continuously provide HIV resistant immune cells throughout the life of a patient. Therefore, hematopoietic stem cell based anti-HIV gene therapy has a great potential to provide a life-long remission of HIV/AIDS by a single treatment. Here, we provide a comprehensive review of the recent progress of developing anti-HIV genes, genetic modification of hematopoietic stem progenitor cells, engraftment and reconstitution of anti-HIV gene modified immune cells, HIV inhibition in vitro and in vivo animal models, and human clinical trials.

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“…Along the same lines, mesenchymal stem cells have been gene edited and converted to CD34+ progenitor cells [27]. Studies in humanized mice suggest that modified hematopoietic stem and progenitor cells can engraft and support multilineage differentiation [28,29]. …”

Section: Suppression or Disruption Of Hiv Co-receptorsmentioning

confidence: 99%

Cell and gene therapy strategies to eradicate HIV reservoirs

Spragg

Feelixge

Jerome

2016

Current Opinion in HIV and AIDS

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Purpose of review Highly active antiretroviral treatment has dramatically improved prognosis for people living with HIV by preventing AIDS-related morbidity and mortality through profound suppression of viral replication. However, a long-lived viral reservoir persists in latently infected cells that harbor replication-competent HIV genomes. If therapy is discontinued, latently infected memory cells inevitably reactivate and produce infectious virus, resulting in viral rebound. The reservoir is the biggest obstacle to a cure of HIV. Recent findings This review summarizes significant advances of the past year in the development of cellular and gene therapies for HIV cure. In particular, we highlight work done on suppression or disruption of HIV co-receptors, vectored delivery of antibodies and antibody-like molecules, T cell therapies, and HIV genome disruption. Summary A number of recent advancements in cellular and gene therapies have emerged at the forefront of HIV cure research, potentially having broad implications for the future of HIV treatment.

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Lentivector Knockdown of CCR5 in Hematopoietic Stem and Progenitor Cells Confers Functional and Persistent HIV-1 Resistance in Humanized Mice

Cited by 32 publications

References 54 publications

Transplantation of gene-modified haematopoietic stem cells: Application and clinical considerations

Transplantation of gene-modified haematopoietic stem cells: Application and clinical considerations

Stem cell-based therapies for HIV/AIDS

Cell and gene therapy strategies to eradicate HIV reservoirs

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