Bone Marrow Gene Therapy for HIV/AIDS

The impact of HIV-1 genetic diversity on the efficacy of a combinatorial RNAi-based gene therapy

(2015) E Herrera-Carrillo et al.   GENE THERAPY

Sustained Delivery of a Broadly Neutralizing Antibody in Nonhuman Primates Confers Long-Term Protection against Simian/Human Immunodeficiency Virus Infection

(2015) Kevin O. Saunders et al.   JOURNAL OF VIROLOGY

Multiplexing Seven miRNA-Based shRNAs to Suppress HIV Replication

(2015) Jang-Gi Choi et al.   MOLECULAR THERAPY

AAV-expressed eCD4-Ig provides durable protection from multiple SHIV challenges

(2015) Matthew R. Gardner et al.   NATURE

Toward optimization of AgoshRNA moleculesthat use a non-canonical RNAi pathway: Variations in the top and bottom base pairs

(2015) Elena Herrera-Carrillo et al.   RNA Biology

Mechanistic insights on the Dicer-independent AGO2-mediated processing of AgoshRNAs

(2015) Ying Poi Liu et al.   RNA Biology

Deep Sequence Analysis of AgoshRNA Processing Reveals 3’ A Addition and Trimming

(2015) Alex Harwig et al.   Molecular Therapy-Nucleic Acids

Zinc Finger Endonuclease TargetingPSIP1Inhibits HIV-1 Integration

(2014) Roger Badia et al.   ANTIMICROBIAL AGENTS AND CHEMOTHERAPY

Towards improved shRNA and miRNA reagents as inhibitors of HIV1 replication

(2014) Ben Berkhout et al.   Future Microbiology

TALEN Knockout of the PSIP1 Gene in Human Cells: Analyses of HIV-1 Replication and Allosteric Integrase Inhibitor Mechanism

(2014) H. J. Fadel et al.   JOURNAL OF VIROLOGY

Vectored immunoprophylaxis protects humanized mice from mucosal HIV transmission

(2014) Alejandro B Balazs et al.   NATURE MEDICINE

Probing the shRNA characteristics that hinder Dicer recognition and consequently allow Ago-mediated processing and AgoshRNA activity

(2014) Elena Herrera-Carrillo et al.   RNA

Proliferation of cells with HIV integrated into cancer genes contributes to persistent infection

(2014) T. A. Wagner et al.   SCIENCE

Specific HIV integration sites are linked to clonal expansion and persistence of infected cells

(2014) F. Maldarelli et al.   SCIENCE

Gene Therapy Strategies to Exploit TRIM Derived Restriction Factors against HIV-1

(2014) Emma Chan et al.   Viruses-Basel

Lack of Detectable HIV-1 Molecular Evolution during Suppressive Antiretroviral Therapy

(2014) Mary F. Kearney et al.   PLoS Pathogens

Antiviral strategies combining antiretroviral drugs with RNAi-mediated attack on HIV-1 and cellular co-factors

(2013) Fatima Boutimah et al.   ANTIVIRAL RESEARCH

Simultaneous zinc-finger nuclease editing of the HIV coreceptors ccr5 and cxcr4 protects CD4+ T cells from HIV-1 infection

(2013) C. A. Didigu et al.   BLOOD

A novel gene therapy strategy using secreted multifunctional anti-HIV proteins to confer protection to gene-modified and unmodified target cells

(2013) A Falkenhagen et al.   GENE THERAPY

A Mutant Tat Protein Provides Strong Protection from HIV-1 Infection in Human CD4+ T Cells

(2013) Ann Apolloni et al.   HUMAN GENE THERAPY

Barriers to a cure for HIV: new ways to target and eradicate HIV-1 reservoirs

(2013) Christine Katlama et al.   LANCET

Introduction of Exogenous T-cell Receptors Into Human Hematopoietic Progenitors Results in Exclusion of Endogenous T-cell Receptor Expression

(2013) Dimitrios N Vatakis et al.   MOLECULAR THERAPY

The Impact of Unprotected T Cells in RNAi-based Gene Therapy for HIV-AIDS

(2013) Elena Herrera-Carrillo et al.   MOLECULAR THERAPY

Efficacy Trial of a DNA/rAd5 HIV-1 Preventive Vaccine

(2013) Scott M. Hammer et al.   NEW ENGLAND JOURNAL OF MEDICINE

Dicer-independent processing of short hairpin RNAs

(2013) Y. P. Liu et al.   NUCLEIC ACIDS RESEARCH

Zinc-finger-nucleases mediate specific and efficient excision of HIV-1 proviral DNA from infected and latently infected human T cells

(2013) Xiying Qu et al.   NUCLEIC ACIDS RESEARCH

Lentiviral Hematopoietic Stem Cell Gene Therapy in Patients with Wiskott-Aldrich Syndrome

(2013) A. Aiuti et al.   SCIENCE

Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy

(2013) A. Biffi et al.   SCIENCE

Preclinical In Vivo Evaluation of the Safety of a Multi-shRNA-based Gene Therapy Against HIV-1

(2013) Mireille Centlivre et al.   Molecular Therapy-Nucleic Acids

Antiviral effects of autologous CD4 T cells genetically modified with a conditionally replicating lentiviral vector expressing long antisense to HIV

(2012) P. Tebas et al.   BLOOD

The Loop Position of shRNAs and Pre-miRNAs Is Critical for the Accuracy of Dicer Processing In Vivo

(2012) Shuo Gu et al.   CELL

T-lymphocyte perturbation following large-scale apheresis and hematopoietic stem cell transplantation in HIV-infected individuals

(2012) Borislav Savkovic et al.   CLINICAL IMMUNOLOGY

Gene therapy as a vaccine for HIV-1

(2012) Ben Berkhout et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

In Vivo Selection of CD4+ T Cells Transduced with a Gamma-Retroviral Vector Expressing a Single-Chain Intrabody Targeting HIV-1 Tat

(2012) Stephen E. Braun et al.   HUMAN GENE THERAPY

Generation of an HIV-1-Resistant Immune System with CD34+ Hematopoietic Stem Cells Transduced with a Triple-Combination Anti-HIV Lentiviral Vector

(2012) J. E. Walker et al.   JOURNAL OF VIROLOGY

Zinc-finger Nuclease Editing of Human cxcr4 Promotes HIV-1 CD4+ T Cell Resistance and Enrichment

(2012) Jinyun Yuan et al.   MOLECULAR THERAPY

Differential integrity of TALE nuclease genes following adenoviral and lentiviral vector gene transfer into human cells

(2012) Maarten Holkers et al.   NUCLEIC ACIDS RESEARCH

microRNAs associated with the different human Argonaute proteins

(2012) A. Dueck et al.   NUCLEIC ACIDS RESEARCH

Decade-Long Safety and Function of Retroviral-Modified Chimeric Antigen Receptor T Cells

(2012) J. Scholler et al.   Science Translational Medicine

Directed HIV-1 Evolution of Protease Inhibitor Resistance by Second-Generation Short Hairpin RNAs

(2011) Nick C. T. Schopman et al.   ANTIMICROBIAL AGENTS AND CHEMOTHERAPY

Treatment intensification with raltegravir in subjects with sustained HIV-1 viraemia suppression: a randomized 48-week study

(2011) Josep M Llibre et al.   ANTIVIRAL THERAPY

A competitive cell growth assay for the detection of subtle effects of gene transduction on cell proliferation

(2011) J J M Eekels et al.   GENE THERAPY

Current progress in the development of RNAi-based therapeutics for HIV-1

(2011) J Zhou et al.   GENE THERAPY

Safety and efficacy of the HVTN 503/Phambili Study of a clade-B-based HIV-1 vaccine in South Africa: a double-blind, randomised, placebo-controlled test-of-concept phase 2b study

(2011) Glenda E Gray et al.   LANCET INFECTIOUS DISEASES

Secreted Antiviral Entry Inhibitory (SAVE) Peptides for Gene Therapy of HIV Infection

(2011) Lisa Egerer et al.   MOLECULAR THERAPY

Antibody-based protection against HIV infection by vectored immunoprophylaxis

(2011) Alejandro B. Balazs et al.   NATURE

A novel TALE nuclease scaffold enables high genome editing activity in combination with low toxicity

(2011) Claudio Mussolino et al.   NUCLEIC ACIDS RESEARCH

An Aptamer-siRNA Chimera Suppresses HIV-1 Viral Loads and Protects from Helper CD4+ T Cell Decline in Humanized Mice

(2011) C. P. Neff et al.   Science Translational Medicine

Engineering HIV-Resistant Human CD4+ T Cells with CXCR4-Specific Zinc-Finger Nucleases

(2011) Craig B. Wilen et al.   PLoS Pathogens

Allogeneic transplantation of CCR5-deficient progenitor cells in a patient with HIV infection: an update after 3 years and the search for patient no. 2

(2010) Gero Hütter et al.   AIDS

Long-term inhibition of HIV-1 replication with RNA interference against cellular co-factors

(2010) Julia J.M. Eekels et al.   ANTIVIRAL RESEARCH

Management of relapsed and refractory classical Hodgkin lymphoma in children and adolescents

(2010) Stephen Daw et al.   BRITISH JOURNAL OF HAEMATOLOGY

Absence of DICER in Monocytes and Its Regulation by HIV-1

(2010) William Coley et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Inhibition of Early Stages of HIV-1 Assembly by INI1/hSNF5 Transdominant Negative Mutant S6

(2010) J. Cano et al.   JOURNAL OF VIROLOGY

Identification of Dominant Negative Human Immunodeficiency Virus Type 1 Vif Mutants That Interfere with the Functional Inactivation of APOBEC3G by Virus-Encoded Vif

(2010) R. C. Walker et al.   JOURNAL OF VIROLOGY

Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo

(2010) Nathalia Holt et al.   NATURE BIOTECHNOLOGY

Deriving four functional anti-HIV siRNAs from a single Pol III-generated transcript comprising two adjacent long hairpin RNA precursors

(2010) Sheena Saayman et al.   NUCLEIC ACIDS RESEARCH

Titers of lentiviral vectors encoding shRNAs and miRNAs are reduced by different mechanisms that require distinct repair strategies

(2010) Y. P. Liu et al.   RNA

RNA-Based Gene Therapy for HIV with Lentiviral Vector-Modified CD34+ Cells in Patients Undergoing Transplantation for AIDS-Related Lymphoma

(2010) D. L. DiGiusto et al.   Science Translational Medicine

The F12-Vif derivative Chim3 inhibits HIV-1 replication in CD4+ T lymphocytes and CD34+-derived macrophages by blocking HIV-1 DNA integration

(2009) S. Porcellini et al.   BLOOD

Lentiviral Delivery of RNAi Effectors Against HIV-1

(2009) Ying Liu et al.   CURRENT TOPICS IN MEDICINAL CHEMISTRY

Foamy combinatorial anti-HIV vectors with MGMTP140K potently inhibit HIV-1 and SHIV replication and mediate selection in vivo

(2009) H-P Kiem et al.   GENE THERAPY

Potent inhibition of HIV-1 by TRIM5-cyclophilin fusion proteins engineered from human components

(2009) Martha R. Neagu et al.   JOURNAL OF CLINICAL INVESTIGATION

Stringent testing identifies highly potent and escape-proof anti-HIV short hairpin RNAs

(2009) Karin J. von Eije et al.   JOURNAL OF GENE MEDICINE

Combinatorial RNAi Against HIV-1 Using Extended Short Hairpin RNAs

(2009) Ying Poi Liu et al.   MOLECULAR THERAPY

Phase 2 gene therapy trial of an anti-HIV ribozyme in autologous CD34+ cells

(2009) Ronald T Mitsuyasu et al.   NATURE MEDICINE

Long-Term Control of HIV byCCR5Delta32/Delta32 Stem-Cell Transplantation

(2009) Gero Hütter et al.   NEW ENGLAND JOURNAL OF MEDICINE

RNA polymerase III can drive polycistronic expression of functional interfering RNAs designed to resemble microRNAs

(2009) L. L. Snyder et al.   NUCLEIC ACIDS RESEARCH

Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy

(2009) N. Cartier et al.   SCIENCE

Recruitment of a SAP18-HDAC1 Complex into HIV-1 Virions and Its Requirement for Viral Replication

(2009) Masha Sorin et al.   PLoS Pathogens

Probing the sequence space available for HIV-1 evolution

(2008) Olivier ter Brake et al.   AIDS

Human Immunodeficiency Virus Type 1 Restriction by Human–Rhesus Chimeric Tripartite Motif 5α (TRIM 5α) in CD34+ Cell-Derived Macrophages In Vitro and in T Cells In Vivo in Severe Combined Immunodeficient (SCID-hu) Mice Transplanted with Human Fetal Tissue

(2008) Joseph Anderson et al.   HUMAN GENE THERAPY

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

(2008) Salima Hacein-Bey-Abina et al.   JOURNAL OF CLINICAL INVESTIGATION

Overcoming resistance to existing therapies in HIV-infected patients: The role of new antiretroviral drugs

(2008) Carlo-Federico Perno et al.   JOURNAL OF MEDICAL VIROLOGY

Lentiviral Vectors Encoding Human Immunodeficiency Virus Type 1 (HIV-1)-Specific T-Cell Receptor Genes Efficiently Convert Peripheral Blood CD8 T Lymphocytes into Cytotoxic T Lymphocytes with Potent In Vitro and In Vivo HIV-1-Specific Inhibitory Activity

(2008) A. Joseph et al.   JOURNAL OF VIROLOGY

Lentiviral Vector Design for Multiple shRNA Expression and Durable HIV-1 Inhibition

(2008) Olivier ter Brake et al.   MOLECULAR THERAPY

Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleases

(2008) Elena E Perez et al.   NATURE BIOTECHNOLOGY

Inhibition of HIV-1 by multiple siRNAs expressed from a single microRNA polycistron

(2008) Ying Poi Liu et al.   NUCLEIC ACIDS RESEARCH

Selective translational repression of HIV-1 RNA by Sam68DeltaC occurs by altering PABP1 binding to unspliced viral RNA

(2008) Kim Marsh et al.   Retrovirology