Potential mechanisms for cell-based gene therapy to treat HIV/AIDS

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

Unravelling the structural and mechanistic basis of CRISPR–Cas systems

(2014) John van der Oost et al.   NATURE REVIEWS MICROBIOLOGY

Gene Editing of CCR5 in Autologous CD4 T Cells of Persons Infected with HIV

(2014) Pablo Tebas et al.   NEW ENGLAND JOURNAL OF 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

The early spread and epidemic ignition of HIV-1 in human populations

(2014) N. R. Faria et al.   SCIENCE

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

(2014) Emma Chan et al.   Viruses-Basel

A Quantitative Comparison of Anti-HIV Gene Therapy Delivered to Hematopoietic Stem Cells versus CD4+ T Cells

(2014) Borislav Savkovic et al.   PLoS Computational Biology

CD4+ T Cells Modified by the Endoribonuclease MazF Are Safe and Can Persist in SHIV-infected Rhesus Macaques

(2014) Naoki Saito et al.   Molecular Therapy-Nucleic Acids

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

(2013) Ann Apolloni et al.   HUMAN GENE THERAPY

Efficient Clinical Scale Gene Modification via Zinc Finger Nuclease–Targeted Disruption of the HIV Co-receptor CCR5

(2013) Dawn A. Maier et al.   HUMAN GENE THERAPY

The Many Lives of CTIP2: From AIDS to Cancer and Cardiac Hypertrophy

(2013) Valentin Le Douce et al.   JOURNAL OF CELLULAR PHYSIOLOGY

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

Myeloid/Microglial Driven Autologous Hematopoietic Stem Cell Gene Therapy Corrects a Neuronopathic Lysosomal Disease

(2013) Ana Sergijenko 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

Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease

(2013) Seung Woo Cho et al.   NATURE BIOTECHNOLOGY

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

p21-mediated RNR2 repression restricts HIV-1 replication in macrophages by inhibiting dNTP biosynthesis pathway

(2013) A. Allouch et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Cell-associated HIV RNA: a dynamic biomarker of viral persistence

(2013) Alexander O Pasternak et al.   Retrovirology

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

RNA-Guided Human Genome Engineering via Cas9

(2013) P. Mali et al.   SCIENCE

Harnessing the CRISPR/Cas9 system to disrupt latent HIV-1 provirus

(2013) Hirotaka Ebina et al.   Scientific Reports

Highly Significant Antiviral Activity of HIV-1 LTR-Specific Tre-Recombinase in Humanized Mice

(2013) Ilona Hauber et al.   PLoS Pathogens

Post-Treatment HIV-1 Controllers with a Long-Term Virological Remission after the Interruption of Early Initiated Antiretroviral Therapy ANRS VISCONTI Study

(2013) Asier Sáez-Cirión et al.   PLoS Pathogens

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

Design of modified U1i molecules against HIV-1 RNA

(2012) Stefanie A. Knoepfel et al.   ANTIVIRAL RESEARCH

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

Hematopoietic-Stem-Cell-Based Gene Therapy for HIV Disease

(2012) Hans-Peter Kiem et al.   Cell Stem Cell

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

(2012) Borislav Savkovic et al.   CLINICAL IMMUNOLOGY

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

Achieving a cure for HIV infection: do we have reasons to be optimistic?

(2012) V. Le Douce et al.   JOURNAL OF ANTIMICROBIAL CHEMOTHERAPY

SHAPE-directed Discovery of Potent shRNA Inhibitors of HIV-1

(2012) Justin T Low et al.   MOLECULAR THERAPY

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

(2012) Jinyun Yuan et al.   MOLECULAR THERAPY

SAMHD1 restricts the replication of human immunodeficiency virus type 1 by depleting the intracellular pool of deoxynucleoside triphosphates

(2012) Hichem Lahouassa et al.   NATURE IMMUNOLOGY

SAMHD1 restricts HIV-1 infection in resting CD4+ T cells

(2012) Hanna-Mari Baldauf et al.   NATURE MEDICINE

TALENs: a widely applicable technology for targeted genome editing

(2012) J. Keith Joung et al.   NATURE REVIEWS MOLECULAR CELL BIOLOGY

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

(2012) Maarten Holkers et al.   NUCLEIC ACIDS RESEARCH

A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity

(2012) M. Jinek et al.   SCIENCE

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

HIV-1 Utilizes the CXCR4 Chemokine Receptor to Infect Multipotent Hematopoietic Stem and Progenitor Cells

(2011) Christoph C. Carter et al.   Cell Host & Microbe

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

HIV-1 restriction factor SAMHD1 is a deoxynucleoside triphosphate triphosphohydrolase

(2011) David C. Goldstone et al.   NATURE

LSD1 cooperates with CTIP2 to promote HIV-1 transcriptional silencing

(2011) V. Le Douce et al.   NUCLEIC ACIDS RESEARCH

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

(2011) Claudio Mussolino et al.   NUCLEIC ACIDS RESEARCH

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

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

Gene Therapy Strategies: Can We Eradicate HIV?

(2011) Jan van Lunzen et al.   Current HIV/AIDS Reports

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

XanthomonasAvrBs3 Family-Type III Effectors: Discovery and Function

(2010) Jens Boch et al.   Annual Review of Phytopathology

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

Inhibition of HIV-1 by Fusion Inhibitors

(2010) Dirk Eggink et al.   CURRENT PHARMACEUTICAL DESIGN

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

Lethal graft-versus-host disease in mouse models of T cell receptor gene therapy

(2010) Gavin M Bendle et al.   NATURE MEDICINE

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

Anticipating and blocking HIV-1 escape by second generation antiviral shRNAs

(2010) Nick CT Schopman et al.   Retrovirology

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

Computational Models of HIV-1 Resistance to Gene Therapy Elucidate Therapy Design Principles

(2010) Sharon Aviran et al.   PLoS Computational Biology

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

Detailed Mechanistic Insights into HIV-1 Sensitivity to Three Generations of Fusion Inhibitors

(2009) Dirk Eggink et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

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

Mathematical modelling of the impact of haematopoietic stem cell-delivered gene therapy for HIV

(2009) John M. Murray 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

Adoptive immunotherapy for cancer: the next generation of gene-engineered immune cells

(2009) L. J. Berry et al.   TISSUE ANTIGENS

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

(2009) Masha Sorin et al.   PLoS Pathogens

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

Highly Sensitive Methods Based on Seminested Real-Time Reverse Transcription-PCR for Quantitation of Human Immunodeficiency Virus Type 1 Unspliced and Multiply Spliced RNA and Proviral DNA

(2008) A. O. Pasternak et al.   JOURNAL OF CLINICAL MICROBIOLOGY

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

Control of HIV-1 immune escape by CD8 T cells expressing enhanced T-cell receptor

(2008) Angel Varela-Rohena et al.   NATURE MEDICINE

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