Newer gene editing technologies toward HIV gene therapy.

PubWeight™: 1.07‹?› | Rank: Top 10%

🔗 View Article (PMC 3856413)

Published in Viruses on November 14, 2013

Authors

N Manjunath1, Guohua Yi, Ying Dang, Premlata Shankar

Author Affiliations

1: Center of Excellence in Infectious Disease, Department of Biomedical Sciences, Paul L. Foster School of Medicine, Texas Tech University Health Sciences Center, El Paso, TX 79905, USA. Manjunath.swamy@ttuhsc.edu.

Associated clinical trials:

Autologous T-Cells Genetically Modified at the CCR5 Gene by Zinc Finger Nucleases SB-728 for HIV (Zinc-Finger) | NCT00842634

Phase 1 Dose Escalation Study of Autologous T-cells Genetically Modified at the CCR5 Gene by Zinc Finger Nucleases in HIV-Infected Patients | NCT01044654

Study of Autologous T-cells Genetically Modified at the CCR5 Gene by Zinc Finger Nucleases in HIV-Infected Subjects | NCT01252641

Articles citing this

RNA-directed gene editing specifically eradicates latent and prevents new HIV-1 infection. Proc Natl Acad Sci U S A (2014) 2.32

Gene therapy for the nervous system: challenges and new strategies. Neurotherapeutics (2014) 0.98

Detection of treatment-resistant infectious HIV after genome-directed antiviral endonuclease therapy. Antiviral Res (2015) 0.97

CCR5 Gene Editing of Resting CD4(+) T Cells by Transient ZFN Expression From HIV Envelope Pseudotyped Nonintegrating Lentivirus Confers HIV-1 Resistance in Humanized Mice. Mol Ther Nucleic Acids (2014) 0.96

Genome editing strategies: potential tools for eradicating HIV-1/AIDS. J Neurovirol (2015) 0.87

The CRISPR/Cas9 genome editing methodology as a weapon against human viruses. Discov Med (2015) 0.86

HIV Excision Utilizing CRISPR/Cas9 Technology: Attacking the Proviral Quasispecies in Reservoirs to Achieve a Cure. MOJ Immunol (2014) 0.85

TALEN gene editing takes aim on HIV. Hum Genet (2016) 0.84

Pervasive Genotypic Mosaicism in Founder Mice Derived from Genome Editing through Pronuclear Injection. PLoS One (2015) 0.82

Hematopoietic stem cell transplantation for HIV cure. J Clin Invest (2016) 0.81

Zinc finger endonuclease targeting PSIP1 inhibits HIV-1 integration. Antimicrob Agents Chemother (2014) 0.81

Engineering T Cells to Functionally Cure HIV-1 Infection. Mol Ther (2015) 0.81

HIV-1 Latency and Eradication: Past, Present and Future. Curr HIV Res (2016) 0.80

Lentivirus pre-packed with Cas9 protein for safer gene editing. Gene Ther (2016) 0.80

In Vivo Excision of HIV-1 Provirus by saCas9 and Multiplex Single-Guide RNAs in Animal Models. Mol Ther (2017) 0.79

Recent advances in RNAi-based strategies for therapy and prevention of HIV-1/AIDS. Adv Drug Deliv Rev (2016) 0.78

Development of Lentiviral Vectors Simultaneously Expressing Multiple siRNAs Against CCR5, vif and tat/rev Genes for an HIV-1 Gene Therapy Approach. Mol Ther Nucleic Acids (2016) 0.76

Gene Editing for Treatment of Neurological Infections. Neurotherapeutics (2016) 0.76

Search for a cure for chronic hepatitis B infection: How close are we? World J Hepatol (2015) 0.75

Inhibition of HIV-1 Viral Infection by an Engineered CRISPR Csy4 RNA Endoribonuclease. PLoS One (2015) 0.75

Perspectives of Genome-Editing Technologies for HIV Therapy. Curr HIV Res (2016) 0.75

Using intron splicing trick for preferential gene expression in transduced cells: an approach for suicide gene therapy. Cancer Gene Ther (2015) 0.75

Targeting CCR5 for anti-HIV research. Eur J Clin Microbiol Infect Dis (2014) 0.75

The therapeutic landscape of HIV-1 via genome editing. AIDS Res Ther (2017) 0.75

Articles cited by this

Multiplex genome engineering using CRISPR/Cas systems. Science (2013) 55.53

RNA-guided human genome engineering via Cas9. Science (2013) 48.29

A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity. Science (2012) 48.17

A TALE nuclease architecture for efficient genome editing. Nat Biotechnol (2010) 26.47

A simplified system for generating recombinant adenoviruses. Proc Natl Acad Sci U S A (1998) 24.58

Site-directed mutagenesis by gene targeting in mouse embryo-derived stem cells. Cell (1987) 20.47

Disruption of the proto-oncogene int-2 in mouse embryo-derived stem cells: a general strategy for targeting mutations to non-selectable genes. Nature (1988) 20.36

DNA targeting specificity of RNA-guided Cas9 nucleases. Nat Biotechnol (2013) 19.99

Genome editing with engineered zinc finger nucleases. Nat Rev Genet (2010) 18.88

High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells. Nat Biotechnol (2013) 18.01

Homozygous defect in HIV-1 coreceptor accounts for resistance of some multiply-exposed individuals to HIV-1 infection. Cell (1996) 17.81

Evolution and classification of the CRISPR-Cas systems. Nat Rev Microbiol (2011) 17.11

Zinc finger-DNA recognition: crystal structure of a Zif268-DNA complex at 2.1 A. Science (1991) 15.97

Resistance to HIV-1 infection in caucasian individuals bearing mutant alleles of the CCR-5 chemokine receptor gene. Nature (1996) 15.91

Genetic restriction of HIV-1 infection and progression to AIDS by a deletion allele of the CKR5 structural gene. Hemophilia Growth and Development Study, Multicenter AIDS Cohort Study, Multicenter Hemophilia Cohort Study, San Francisco City Cohort, ALIVE Study. Science (1996) 15.63

RNA-guided editing of bacterial genomes using CRISPR-Cas systems. Nat Biotechnol (2013) 15.51

Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleases. Nat Biotechnol (2008) 14.75

Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease. Nat Biotechnol (2013) 14.28

Heritable targeted gene disruption in zebrafish using designed zinc-finger nucleases. Nat Biotechnol (2008) 14.20

Repurposing CRISPR as an RNA-guided platform for sequence-specific control of gene expression. Cell (2013) 13.43

Double nicking by RNA-guided CRISPR Cas9 for enhanced genome editing specificity. Cell (2013) 13.37

FLASH assembly of TALENs for high-throughput genome editing. Nat Biotechnol (2012) 12.97

Efficient construction of sequence-specific TAL effectors for modulating mammalian transcription. Nat Biotechnol (2011) 12.86

Latent infection of CD4+ T cells provides a mechanism for lifelong persistence of HIV-1, even in patients on effective combination therapy. Nat Med (1999) 12.68

Chimeric nucleases stimulate gene targeting in human cells. Science (2003) 12.12

ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering. Trends Biotechnol (2013) 11.58

CAS9 transcriptional activators for target specificity screening and paired nickases for cooperative genome engineering. Nat Biotechnol (2013) 11.35

Targeted gene inactivation in zebrafish using engineered zinc-finger nucleases. Nat Biotechnol (2008) 10.29

The mechanism of double-strand DNA break repair by the nonhomologous DNA end-joining pathway. Annu Rev Biochem (2010) 9.80

Long-term follow-up studies confirm the stability of the latent reservoir for HIV-1 in resting CD4+ T cells. Nat Med (2003) 9.75

Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery. Nat Biotechnol (2007) 9.23

Targeted chromosomal cleavage and mutagenesis in Drosophila using zinc-finger nucleases. Genetics (2002) 9.09

Targeted genome editing in human cells with zinc finger nucleases constructed via modular assembly. Genome Res (2009) 8.69

Heritable gene targeting in zebrafish using customized TALENs. Nat Biotechnol (2011) 8.34

A novel TALE nuclease scaffold enables high genome editing activity in combination with low toxicity. Nucleic Acids Res (2011) 7.93

The knockout mouse project. Nat Genet (2004) 7.80

Xanthomonas AvrBs3 family-type III effectors: discovery and function. Annu Rev Phytopathol (2010) 7.70

Progress and prospects: zinc-finger nucleases as gene therapy agents. Gene Ther (2008) 7.28

High-frequency modification of plant genes using engineered zinc-finger nucleases. Nature (2009) 7.19

Targeted gene disruption in somatic zebrafish cells using engineered TALENs. Nat Biotechnol (2011) 7.05

TALENs: a widely applicable technology for targeted genome editing. Nat Rev Mol Cell Biol (2012) 7.01

Revealing off-target cleavage specificities of zinc-finger nucleases by in vitro selection. Nat Methods (2011) 6.90

A transcription activator-like effector toolbox for genome engineering. Nat Protoc (2012) 6.81

Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo. Nat Biotechnol (2010) 6.55

An unbiased genome-wide analysis of zinc-finger nuclease specificity. Nat Biotechnol (2011) 6.44

Enhancing zinc-finger-nuclease activity with improved obligate heterodimeric architectures. Nat Methods (2010) 6.00

Mitotic homologous recombination maintains genomic stability and suppresses tumorigenesis. Nat Rev Mol Cell Biol (2010) 5.35

FokI dimerization is required for DNA cleavage. Proc Natl Acad Sci U S A (1998) 5.31

The mechanism of human nonhomologous DNA end joining. J Biol Chem (2007) 5.02

Evidence for the cure of HIV infection by CCR5Δ32/Δ32 stem cell transplantation. Blood (2010) 4.99

Structures of the RNA-guided surveillance complex from a bacterial immune system. Nature (2011) 4.92

Modularly assembled designer TAL effector nucleases for targeted gene knockout and gene replacement in eukaryotes. Nucleic Acids Res (2011) 4.75

Precise genome modification in the crop species Zea mays using zinc-finger nucleases. Nature (2009) 4.71

Targeted disruption of the murine int-1 proto-oncogene resulting in severe abnormalities in midbrain and cerebellar development. Nature (1990) 3.92

Extended follow-up confirms early vaccine-enhanced risk of HIV acquisition and demonstrates waning effect over time among participants in a randomized trial of recombinant adenovirus HIV vaccine (Step Study). J Infect Dis (2012) 3.42

Transcriptional activators of human genes with programmable DNA-specificity. PLoS One (2011) 3.16

Assembly of designer TAL effectors by Golden Gate cloning. PLoS One (2011) 3.05

Custom zinc-finger nucleases for use in human cells. Mol Ther (2005) 3.04

Harnessing the CRISPR/Cas9 system to disrupt latent HIV-1 provirus. Sci Rep (2013) 2.48

A CRISPR/Cas system mediates bacterial innate immune evasion and virulence. Nature (2013) 2.44

Site-specific integration and tailoring of cassette design for sustainable gene transfer. Nat Methods (2011) 2.30

Differential integrity of TALE nuclease genes following adenoviral and lentiviral vector gene transfer into human cells. Nucleic Acids Res (2012) 2.14

Iterative capped assembly: rapid and scalable synthesis of repeat-module DNA such as TAL effectors from individual monomers. Nucleic Acids Res (2012) 2.09

Targeted gene knockout by direct delivery of zinc-finger nuclease proteins. Nat Methods (2012) 2.07

A ligation-independent cloning technique for high-throughput assembly of transcription activator–like effector genes. Nat Biotechnol (2013) 1.98

Advances in targeted genome editing. Curr Opin Chem Biol (2012) 1.97

HIV-1 coreceptor use in triple-class treatment-experienced patients: baseline prevalence, correlates, and relationship to enfuvirtide response. J Infect Dis (2006) 1.70

Zinc-finger nucleases as a novel therapeutic strategy for targeting hepatitis B virus DNAs. Mol Ther (2010) 1.61

Low-dose penile SIVmac251 exposure of rhesus macaques infected with adenovirus type 5 (Ad5) and then immunized with a replication-defective Ad5-based SIV gag/pol/nef vaccine recapitulates the results of the phase IIb step trial of a similar HIV-1 vaccine. J Virol (2011) 1.52

Engineering HIV-resistant human CD4+ T cells with CXCR4-specific zinc-finger nucleases. PLoS Pathog (2011) 1.50

Genomic editing of the HIV-1 coreceptor CCR5 in adult hematopoietic stem and progenitor cells using zinc finger nucleases. Mol Ther (2013) 1.49

Zinc-finger-nucleases mediate specific and efficient excision of HIV-1 proviral DNA from infected and latently infected human T cells. Nucleic Acids Res (2013) 1.48

A maraviroc-resistant HIV-1 with narrow cross-resistance to other CCR5 antagonists depends on both N-terminal and extracellular loop domains of drug-bound CCR5. J Virol (2010) 1.47

Toxicity of antiretroviral therapy and implications for drug development. Nat Rev Drug Discov (2003) 1.42

Design, engineering, and characterization of zinc finger nucleases. Biochem Biophys Res Commun (2005) 1.39

Multiple drug class-wide resistance associated with poorer survival after treatment failure in a cohort of HIV-infected patients. AIDS (2005) 1.37

Gene targeting in the rat: advances and opportunities. Trends Genet (2010) 1.25

Efficient clinical scale gene modification via zinc finger nuclease-targeted disruption of the HIV co-receptor CCR5. Hum Gene Ther (2013) 1.14

Engineering designer transcription activator-like effector nucleases (TALENs) by REAL or REAL-Fast assembly. Curr Protoc Mol Biol (2012) 1.10

Zinc-finger nuclease editing of human cxcr4 promotes HIV-1 CD4(+) T cell resistance and enrichment. Mol Ther (2012) 1.10

Generation of an HIV resistant T-cell line by targeted "stacking" of restriction factors. Mol Ther (2013) 1.10

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. AIDS (2011) 1.09

Targeted gene disruption to cure HIV. Curr Opin HIV AIDS (2013) 1.09

Zinc-finger nucleases: how to play two good hands. Nat Methods (2011) 1.08

Generation of CD34+ cells from CCR5-disrupted human embryonic and induced pluripotent stem cells. Hum Gene Ther (2011) 1.05

Gene editing of human embryonic stem cells via an engineered baculoviral vector carrying zinc-finger nucleases. Mol Ther (2011) 0.97

High-titer adenovirus vector production in 293S cell perfusion culture. Biotechnol Prog (2004) 0.97

Safety concerns about CCR5 as an antiviral target. Curr Opin HIV AIDS (2009) 0.91

RNA interference-based therapeutics for human immunodeficiency virus HIV-1 treatment: synthetic siRNA or vector-based shRNA? Expert Opin Biol Ther (2010) 0.87

A novel therapeutic molecule against HTLV-1 infection targeting provirus. Leukemia (2013) 0.85

The 32-base pair deletion of the chemokine receptor 5 gene (CCR5-Delta32) is not associated with primary sclerosing cholangitis in 363 Scandinavian patients. Tissue Antigens (2006) 0.84

Identity of zinc finger nucleases with specificity to herpes simplex virus type II genomic DNA: novel HSV-2 vaccine/therapy precursors. Theor Biol Med Model (2011) 0.82

Proviral HIV-genome-wide and pol-gene specific zinc finger nucleases: usability for targeted HIV gene therapy. Theor Biol Med Model (2011) 0.81

TNF-α levels are not increased in inflamed patients carrying the CCR5 deletion 32. Cytokine (2010) 0.78

Articles by these authors

RNA interference targeting Fas protects mice from fulminant hepatitis. Nat Med (2003) 7.37

Antibody mediated in vivo delivery of small interfering RNAs via cell-surface receptors. Nat Biotechnol (2005) 6.40

siRNA-directed inhibition of HIV-1 infection. Nat Med (2002) 6.36

miRNA profiling of naïve, effector and memory CD8 T cells. PLoS One (2007) 5.43

Transvascular delivery of small interfering RNA to the central nervous system. Nature (2007) 5.21

T cell-specific siRNA delivery suppresses HIV-1 infection in humanized mice. Cell (2008) 4.32

Sustained small interfering RNA-mediated human immunodeficiency virus type 1 inhibition in primary macrophages. J Virol (2003) 2.26

Most antiviral CD8 T cells during chronic viral infection do not express high levels of perforin and are not directly cytotoxic. Blood (2002) 1.94

Lentiviral delivery of short hairpin RNAs. Adv Drug Deliv Rev (2009) 1.65

RNAi-mediated CCR5 silencing by LFA-1-targeted nanoparticles prevents HIV infection in BLT mice. Mol Ther (2009) 1.55

A single siRNA suppresses fatal encephalitis induced by two different flaviviruses. PLoS Med (2006) 1.55

Lentiviral delivery of short hairpin RNAs protects CD4 T cells from multiple clades and primary isolates of HIV. Blood (2005) 1.54

Positive correlation between neovascularization degree of carotid atherosclerosis determined by contrast-enhanced ultrasound and level of serum C-reactive protein. Vasa (2015) 1.38

Targeted delivery of siRNA to macrophages for anti-inflammatory treatment. Mol Ther (2010) 1.28

Moloney leukemia virus 10 (MOV10) protein inhibits retrovirus replication. J Biol Chem (2010) 1.27

Biochemical differentiation of APOBEC3F and APOBEC3G proteins associated with HIV-1 life cycle. J Biol Chem (2006) 1.19

Analysis of human APOBEC3H haplotypes and anti-human immunodeficiency virus type 1 activity. J Virol (2011) 1.19

Interfering with disease: opportunities and roadblocks to harnessing RNA interference. Trends Mol Med (2003) 1.10

The functional CD8 T cell response to HIV becomes type-specific in progressive disease. J Clin Invest (2002) 1.09

Targeted delivery of small interfering RNA to human dendritic cells to suppress dengue virus infection and associated proinflammatory cytokine production. J Virol (2009) 1.06

Interfering antiviral immunity: application, subversion, hope? Trends Immunol (2006) 1.03

APOBEC3G and APOBEC3F require an endogenous cofactor to block HIV-1 replication. PLoS Pathog (2008) 0.99

NFAT5 regulates HIV-1 in primary monocytes via a highly conserved long terminal repeat site. PLoS Pathog (2006) 0.99

Human macrophage and dendritic cell-specific silencing of high-mobility group protein B1 ameliorates sepsis in a humanized mouse model. Proc Natl Acad Sci U S A (2012) 0.94

A novel HIV-1 restriction factor that is biologically distinct from APOBEC3 cytidine deaminases in a human T cell line CEM.NKR. Retrovirology (2009) 0.94

Avoiding the kiss of death: how HIV and other chronic viruses survive. Curr Opin Immunol (2002) 0.93

Improved siRNA/shRNA functionality by mismatched duplex. PLoS One (2011) 0.89

Silencing early viral replication in macrophages and dendritic cells effectively suppresses flavivirus encephalitis. PLoS One (2011) 0.89

Identification of molecular determinants from Moloney leukemia virus 10 homolog (MOV10) protein for virion packaging and anti-HIV-1 activity. J Biol Chem (2011) 0.89

ORF390 of white spot syndrome virus genome is identified as a novel anti-apoptosis gene. Biochem Biophys Res Commun (2004) 0.87

Enhanced induction of HIV-specific cytotoxic T lymphocytes by dendritic cell-targeted delivery of SOCS-1 siRNA. Mol Ther (2010) 0.87

The mitochondrial translocator protein, TSPO, inhibits HIV-1 envelope glycoprotein biosynthesis via the endoplasmic reticulum-associated protein degradation pathway. J Virol (2014) 0.87

RNA interference-based therapeutics for human immunodeficiency virus HIV-1 treatment: synthetic siRNA or vector-based shRNA? Expert Opin Biol Ther (2010) 0.87

Antibody-mediated delivery of siRNAs for anti-HIV therapy. Methods Mol Biol (2011) 0.85

Humanized mice for studying human leukocyte integrins in vivo. Methods Mol Biol (2012) 0.85

Evidence for Vpr-dependent HIV-1 replication in human CD4+ CEM.NKR T-cells. Retrovirology (2012) 0.84

Demonstration of a novel HIV-1 restriction phenotype from a human T cell line. PLoS One (2008) 0.84

Long-term engraftment of human natural T regulatory cells in NOD/SCID IL2rγc(null) mice by expression of human IL-2. PLoS One (2012) 0.84

Single amino acid change in gp41 region of HIV-1 alters bystander apoptosis and CD4 decline in humanized mice. Virol J (2011) 0.82

Effective suppression of HIV-1 by artificial bispecific miRNA targeting conserved sequences with tolerance for wobble base-pairing. Biochem Biophys Res Commun (2008) 0.81

Alkali burn causes aldehyde dehydrogenase 3A1 (ALDH3A1) decrease in mouse cornea. Mol Vis (2004) 0.81

Cross-clade CD8 T-cell responses to HIV(IIIB) and Chinese B' and C/B' viruses in North American and Chinese HIV-seropositive donors. J Acquir Immune Defic Syndr (2004) 0.80

Contrast-enhanced ultrasound improves accurate identification of appendiceal mucinous adenocarcinoma in an old patient: A case report. Medicine (Baltimore) (2016) 0.79

Targeting the HIV entry, assembly and release pathways for anti-HIV gene therapy. Virology (2011) 0.78

A novel in vitro transcription method for producing siRNAs without specific sequence requirements. Mol Biotechnol (2005) 0.76

Targeting DNA vaccines to myeloid cells using a small peptide. Eur J Immunol (2014) 0.76

A homogeneous fluorometric assay platform based on novel synthetic proteins. Biochem Biophys Res Commun (2007) 0.75

[RNase III-prepared short interfering RNAs induce degradation of SARS-coronavirus mRNAs in human cells]. Sheng Wu Gong Cheng Xue Bao (2004) 0.75