(Q37862074)

24 August 2017

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title

State-of-the-art gene-based therapies: the road ahead (English)

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24 August 2017

Mark A Kay

1

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24 August 2017

publication date

6 April 2011

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24 August 2017

published in

Nature Reviews Genetics

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24 August 2017

volume

12

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24 August 2017

issue

5

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24 August 2017

page(s)

316-328

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Genome editing with engineered zinc finger nucleases

24 August 2017

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In vivo gene therapy of hemophilia B: sustained partial correction in factor IX-deficient dogs

31 March 2020

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31 March 2020

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31 March 2020

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31 March 2020

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31 March 2020

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Update on gene therapy for immunodeficiencies

31 March 2020

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Stem-cell gene therapy for the Wiskott-Aldrich syndrome

31 March 2020

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31 March 2020

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Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy

31 March 2020

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Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy

31 March 2020

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Retroviral integration and human gene therapy

31 March 2020

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Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

31 March 2020

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Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy

31 March 2020

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Genome-wide analysis of retroviral DNA integration

31 March 2020

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RNA-based gene therapy for HIV with lentiviral vector-modified CD34(+) cells in patients undergoing transplantation for AIDS-related lymphoma

31 March 2020

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Correction of junctional epidermolysis bullosa by transplantation of genetically modified epidermal stem cells.

31 March 2020

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Inherited epidermolysis bullosa: past, present, and future

31 March 2020

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Gene therapy for recessive dystrophic epidermolysis bullosa

31 March 2020

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Foamy virus vectors for gene transfer

31 March 2020

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Progress towards liver and lung-directed gene therapy with helper-dependent adenoviral vectors

31 March 2020

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The influence of innate and pre-existing immunity on adenovirus therapy

31 March 2020

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Immune response to helper dependent adenoviral mediated liver gene therapy: challenges and prospects.

31 March 2020

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Gene therapy finds its niche

31 March 2020

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The host response to adenovirus, helper-dependent adenovirus, and adeno-associated virus in mouse liver

31 March 2020

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New insights on adenovirus as vaccine vectors

31 March 2020

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Novel adenovirus vector-based vaccines for HIV-1.

31 March 2020

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Overview of STEP and Phambili trial results: two phase IIb test-of-concept studies investigating the efficacy of MRK adenovirus type 5 gag/pol/nef subtype B HIV vaccine

31 March 2020

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In vivo reprogramming of adult pancreatic exocrine cells to beta-cells

31 March 2020

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Pancreatic and duodenal homeobox gene 1 induces expression of insulin genes in liver and ameliorates streptozotocin-induced hyperglycemia

31 March 2020

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Gene therapy for diabetes: reinventing the islet

31 March 2020

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NeuroD-betacellulin gene therapy induces islet neogenesis in the liver and reverses diabetes in mice

31 March 2020

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Adenovirus transduction is required for the correction of diabetes using Pdx-1 or Neurogenin-3 in the liver

31 March 2020

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Chemokine receptor 5 knockout strategies

31 March 2020

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Gene therapy using adeno-associated virus vectors

31 March 2020

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Clinical gene therapy using recombinant adeno-associated virus vectors

31 March 2020

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New recombinant serotypes of AAV vectors.

31 March 2020

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AAV hybrid serotypes: improved vectors for gene delivery

31 March 2020

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Characterization of tissue tropism determinants of adeno-associated virus type 1.

31 March 2020

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Next generation of adeno-associated virus 2 vectors: point mutations in tyrosines lead to high-efficiency transduction at lower doses

31 March 2020

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Engineering and selection of shuffled AAV genomes: a new strategy for producing targeted biological nanoparticles

31 March 2020

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In vitro and in vivo gene therapy vector evolution via multispecies interbreeding and retargeting of adeno-associated viruses

31 March 2020

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Directed evolution of adeno-associated virus yields enhanced gene delivery vectors

31 March 2020

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Frequency and spectrum of genomic integration of recombinant adeno-associated virus serotype 8 vector in neonatal mouse liver

31 March 2020

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Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo

31 March 2020

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Large-scale molecular characterization of adeno-associated virus vector integration in mouse liver

31 March 2020

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AAV vector integration sites in mouse hepatocellular carcinoma.

31 March 2020

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AAV vectors and tumorigenicity

31 March 2020

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Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial

31 March 2020

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Safety and efficacy of gene transfer for Leber's congenital amaurosis

31 March 2020

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Suppression and replacement gene therapy for autosomal dominant disease in a murine model of dominant retinitis pigmentosa

31 March 2020

1 reference

Improved retinal function in a mouse model of dominant retinitis pigmentosa following AAV-delivered gene therapy

31 March 2020

1 reference

SERCA2a gene transfer enhances eNOS expression and activity in endothelial cells

31 March 2020

1 reference

Design of a phase 1/2 trial of intracoronary administration of AAV1/SERCA2a in patients with heart failure.

31 March 2020

1 reference

AAV-1-mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells

31 March 2020

1 reference

Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy

31 March 2020

1 reference

AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B.

31 March 2020

1 reference

Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector.

31 March 2020

1 reference

Gene therapy for muscle disease

31 March 2020

1 reference

Sustained AAV-mediated dystrophin expression in a canine model of Duchenne muscular dystrophy with a brief course of immunosuppression

31 March 2020

1 reference

A phase I study of aromatic L-amino acid decarboxylase gene therapy for Parkinson's disease

31 March 2020

1 reference

Biodistribution and safety assessment of AAV2-GAD following intrasubthalamic injection in the rat.

31 March 2020

1 reference

Safety and tolerability of putaminal AADC gene therapy for Parkinson disease

31 March 2020

1 reference

Results from a phase I safety trial of hAADC gene therapy for Parkinson disease

31 March 2020

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Eight years of clinical improvement in MPTP-lesioned primates after gene therapy with AAV2-hAADC.

31 March 2020

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Controlled dissemination of AAV vectors in the primate brain

31 March 2020

1 reference

Real-time MR imaging of adeno-associated viral vector delivery to the primate brain

31 March 2020

1 reference

Efficient gene therapy-based method for the delivery of therapeutics to primate cortex

31 March 2020

1 reference

Prevention of spontaneous bleeding in dogs with haemophilia A and haemophilia B.

31 March 2020

1 reference

Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

31 March 2020

1 reference

Hemophilia gene therapy: a Holy Grail found

31 March 2020

1 reference

Non-viral vectors in cystic fibrosis gene therapy: recent developments and future prospects

31 March 2020

1 reference

Current status of polymeric gene delivery systems

31 March 2020

1 reference

Nonviral gene delivery: principle, limitations, and recent progress

31 March 2020

1 reference

CpG-free plasmids confer reduced inflammation and sustained pulmonary gene expression.

31 March 2020

1 reference

Progress and prospects: nuclear import of nonviral vectors

31 March 2020

1 reference

Natural polymers for gene delivery and tissue engineering

31 March 2020

1 reference

Gene therapy progress and prospects: hydrodynamic gene delivery

31 March 2020

1 reference

Hydrodynamics-based transfection in animals by systemic administration of plasmid DNA.

31 March 2020

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Minimally invasive and selective hydrodynamic gene therapy of liver segments in the pig and human

31 March 2020

1 reference

DNA electrotransfer: its principles and an updated review of its therapeutic applications

31 March 2020

1 reference

Minicircle DNA vectors devoid of bacterial DNA result in persistent and high-level transgene expression in vivo

31 March 2020

1 reference

Silencing of episomal transgene expression in liver by plasmid bacterial backbone DNA is independent of CpG methylation

31 March 2020

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A robust system for production of minicircle DNA vectors

31 March 2020

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Progress and prospects: the design and production of plasmid vectors.

31 March 2020

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The expanding universe of transposon technologies for gene and cell engineering.

31 March 2020

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Site-directed transposon integration in human cells

31 March 2020

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Hyperactive sleeping beauty transposase enables persistent phenotypic correction in mice and a canine model for hemophilia B.

31 March 2020

1 reference

The phiC31 integrase system for gene therapy

31 March 2020

1 reference

Integration specificity of phage phiC31 integrase in the human genome

31 March 2020

1 reference

A direct comparison of two nonviral gene therapy vectors for somatic integration: in vivo evaluation of the bacteriophage integrase phiC31 and the Sleeping Beauty transposase

31 March 2020

1 reference

Prevalence of serum IgG and neutralizing factors against adeno-associated virus (AAV) types 1, 2, 5, 6, 8, and 9 in the healthy population: implications for gene therapy using AAV vectors

31 March 2020

1 reference

Update on the prevalence of serum antibodies (IgG and IgM) to adeno-associated virus (AAV).

31 March 2020

1 reference

Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state

31 March 2020

1 reference

Endogenous microRNA regulation suppresses transgene expression in hematopoietic lineages and enables stable gene transfer

31 March 2020

1 reference

Adenoviral vector-based strategies for cancer therapy

31 March 2020

1 reference

Gene therapy with human and mouse T-cell receptors mediates cancer regression and targets normal tissues expressing cognate antigen

31 March 2020

1 reference

Virus-specific T cells engineered to coexpress tumor-specific receptors: persistence and antitumor activity in individuals with neuroblastoma

31 March 2020

1 reference

Gefitinib or carboplatin-paclitaxel in pulmonary adenocarcinoma

31 March 2020

1 reference

Treatment of cancer patients with a serotype 5/3 chimeric oncolytic adenovirus expressing GMCSF.

31 March 2020

1 reference

GM-CSF-armed, replication-competent viruses for cancer

31 March 2020

1 reference

Therapeutic microRNA delivery suppresses tumorigenesis in a murine liver cancer model

31 March 2020

1 reference

Enhanced exon-skipping induced by U7 snRNA carrying a splicing silencer sequence: Promising tool for DMD therapy

31 March 2020

1 reference

AAV vector-mediated RNAi of mutant huntingtin expression is neuroprotective in a novel genetic rat model of Huntington's disease

31 March 2020

1 reference

RNAi suppresses polyglutamine-induced neurodegeneration in a model of spinocerebellar ataxia.

31 March 2020

1 reference

Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways

31 March 2020

1 reference

Argonaute proteins are key determinants of RNAi efficacy, toxicity, and persistence in the adult mouse liver

31 March 2020

1 reference

Current prospects for RNA interference-based therapies

31 March 2020

1 reference

12 December 2020

Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors

1 reference

12 December 2020

Fiber-modified adenoviruses for targeted gene therapy

1 reference

12 December 2020

The mechanism of naked DNA uptake and expression

1 reference

12 December 2020

Hydrodynamics-based gene delivery

1 reference

12 December 2020

Ex vivo gene transfer and correction for cell-based therapies

1 reference

12 December 2020

Adeno-associated virus for the treatment of muscle diseases: toward clinical trials.

1 reference

12 December 2020

Adeno-associated virus vector integration

1 reference

12 December 2020

Experiments in transgenic mice show that hepatocytes are the source for postnatal liver growth and do not stream

1 reference

12 December 2020

New serotypes of adenoviral vectors

1 reference

12 December 2020

Suppression of lung adenocarcinoma progression by Nkx2-1

1 reference

12 December 2020

Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges

1 reference

12 December 2020

Gene targeted therapeutics for liver disease in alpha-1 antitrypsin deficiency

1 reference

12 December 2020

Engineering and Selection of Shuffled AAV Genomes: A New Strategy for Producing Targeted Biological Nanoparticles

1 reference

7 January 2021

inferred from DOI database lookup

Identifiers

DOI

10.1038/NRG2971

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Dimensions Publication ID

24 August 2017

1030150959

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