Bone Marrow Gene Therapy for HIV/AIDS

Bone marrow gene therapy remains an attractive option for treating chronic immunological diseases, including acquired immunodeficiency syndrome (AIDS) caused by human immunodeficiency virus (HIV). This technology combines the differentiation and expansion capacity of hematopoietic stem cells (HSCs)...

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Detalles Bibliográficos
Autores: Herrera, Elena, Berkhout, Ben
Tipo de recurso: artículo
Estado:Versión publicada
Fecha de publicación:2015
País:España
Institución:Consejo Superior de Investigaciones Científicas (CSIC)
Repositorio:DIGITAL.CSIC. Repositorio Institucional del CSIC
OAI Identifier:oai:digital.csic.es:10261/415887
Acceso en línea:http://hdl.handle.net/10261/415887
https://api.elsevier.com/content/abstract/scopus_id/84937413297
Access Level:acceso abierto
Palabra clave:HIV-1
RNAi
Antiviral
Bone marrow
Gene therapy
Hematopoietic stem cell (HSC)
Lentiviral vector
Virus
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spelling Bone Marrow Gene Therapy for HIV/AIDSHerrera, ElenaBerkhout, BenHIV-1RNAiAntiviralBone marrowGene therapyHematopoietic stem cell (HSC)Lentiviral vectorVirusBone marrow gene therapy remains an attractive option for treating chronic immunological diseases, including acquired immunodeficiency syndrome (AIDS) caused by human immunodeficiency virus (HIV). This technology combines the differentiation and expansion capacity of hematopoietic stem cells (HSCs) with long-term expression of therapeutic transgenes using integrating vectors. In this review we summarize the potential of bone marrow gene therapy for the treatment of HIV/AIDS. A broad range of antiviral strategies are discussed, with a particular focus on RNA-based therapies. The idea is to develop a durable gene therapy that lasts the life span of the infected individual, thus contrasting with daily drug regimens to suppress the virus. Different approaches have been proposed to target either the virus or cellular genes encoding co-factors that support virus replication. Some of these therapies have been tested in clinical trials, providing proof of principle that gene therapy is a safe option for treating HIV/AIDS. In this review several topics are discussed, ranging from the selection of the antiviral molecule and the viral target to the optimal vector system for gene delivery and the setup of appropriate preclinical test systems. The molecular mechanisms used to formulate a cure for HIV infection are described, including the latest antiviral strategies and their therapeutic applications. Finally, a potent combination of anti-HIV genes based on our own research program is described.This work was supported by the Nederlandse Organisatie voor Wetenschappelijk Onderzoek-Chemische Wetenschappen (NWO-CW, Top Grant) and Zorg Onderzoek Nederland-Medische Wetenschappen (ZonMw, Translational Gene Therapy Grant).Peer reviewedMultidisciplinary Digital Publishing InstituteDutch Research CouncilZonMwHerrera, Elena [0000-0001-9986-8552]202620262015info:eu-repo/semantics/articlehttp://purl.org/coar/resource_type/c_dcae04bcPublisher's versioninfo:eu-repo/semantics/publishedVersionapplication/pdfhttp://hdl.handle.net/10261/415887https://api.elsevier.com/content/abstract/scopus_id/84937413297reponame:DIGITAL.CSIC. Repositorio Institucional del CSICinstname:Consejo Superior de Investigaciones Científicas (CSIC)Ingléshttps://doi.org/10.3390/v7072804Noinfo:eu-repo/semantics/openAccessoai:digital.csic.es:10261/4158872026-05-22T06:33:51Z
dc.title.none.fl_str_mv Bone Marrow Gene Therapy for HIV/AIDS
title Bone Marrow Gene Therapy for HIV/AIDS
spellingShingle Bone Marrow Gene Therapy for HIV/AIDS
Herrera, Elena
HIV-1
RNAi
Antiviral
Bone marrow
Gene therapy
Hematopoietic stem cell (HSC)
Lentiviral vector
Virus
title_short Bone Marrow Gene Therapy for HIV/AIDS
title_full Bone Marrow Gene Therapy for HIV/AIDS
title_fullStr Bone Marrow Gene Therapy for HIV/AIDS
title_full_unstemmed Bone Marrow Gene Therapy for HIV/AIDS
title_sort Bone Marrow Gene Therapy for HIV/AIDS
dc.creator.none.fl_str_mv Herrera, Elena
Berkhout, Ben
author Herrera, Elena
author_facet Herrera, Elena
Berkhout, Ben
author_role author
author2 Berkhout, Ben
author2_role author
dc.contributor.none.fl_str_mv Dutch Research Council
ZonMw
Herrera, Elena [0000-0001-9986-8552]
dc.subject.none.fl_str_mv HIV-1
RNAi
Antiviral
Bone marrow
Gene therapy
Hematopoietic stem cell (HSC)
Lentiviral vector
Virus
topic HIV-1
RNAi
Antiviral
Bone marrow
Gene therapy
Hematopoietic stem cell (HSC)
Lentiviral vector
Virus
description Bone marrow gene therapy remains an attractive option for treating chronic immunological diseases, including acquired immunodeficiency syndrome (AIDS) caused by human immunodeficiency virus (HIV). This technology combines the differentiation and expansion capacity of hematopoietic stem cells (HSCs) with long-term expression of therapeutic transgenes using integrating vectors. In this review we summarize the potential of bone marrow gene therapy for the treatment of HIV/AIDS. A broad range of antiviral strategies are discussed, with a particular focus on RNA-based therapies. The idea is to develop a durable gene therapy that lasts the life span of the infected individual, thus contrasting with daily drug regimens to suppress the virus. Different approaches have been proposed to target either the virus or cellular genes encoding co-factors that support virus replication. Some of these therapies have been tested in clinical trials, providing proof of principle that gene therapy is a safe option for treating HIV/AIDS. In this review several topics are discussed, ranging from the selection of the antiviral molecule and the viral target to the optimal vector system for gene delivery and the setup of appropriate preclinical test systems. The molecular mechanisms used to formulate a cure for HIV infection are described, including the latest antiviral strategies and their therapeutic applications. Finally, a potent combination of anti-HIV genes based on our own research program is described.
publishDate 2015
dc.date.none.fl_str_mv 2015
2026
2026
dc.type.none.fl_str_mv info:eu-repo/semantics/article
http://purl.org/coar/resource_type/c_dcae04bc
Publisher's version
info:eu-repo/semantics/publishedVersion
format article
status_str publishedVersion
dc.identifier.none.fl_str_mv http://hdl.handle.net/10261/415887
https://api.elsevier.com/content/abstract/scopus_id/84937413297
url http://hdl.handle.net/10261/415887
https://api.elsevier.com/content/abstract/scopus_id/84937413297
dc.language.none.fl_str_mv Inglés
language_invalid_str_mv Inglés
dc.relation.none.fl_str_mv https://doi.org/10.3390/v7072804
No
dc.rights.none.fl_str_mv info:eu-repo/semantics/openAccess
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv application/pdf
dc.publisher.none.fl_str_mv Multidisciplinary Digital Publishing Institute
publisher.none.fl_str_mv Multidisciplinary Digital Publishing Institute
dc.source.none.fl_str_mv reponame:DIGITAL.CSIC. Repositorio Institucional del CSIC
instname:Consejo Superior de Investigaciones Científicas (CSIC)
instname_str Consejo Superior de Investigaciones Científicas (CSIC)
reponame_str DIGITAL.CSIC. Repositorio Institucional del CSIC
collection DIGITAL.CSIC. Repositorio Institucional del CSIC
repository.name.fl_str_mv
repository.mail.fl_str_mv
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