State-of-the-art gene-based therapies: the road ahead (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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Gene therapy finds its niche

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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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31 March 2020

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31 March 2020

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31 March 2020

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AAV hybrid serotypes: improved vectors for gene delivery

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

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

31 March 2020

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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

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

31 March 2020

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SERCA2a gene transfer enhances eNOS expression and activity in endothelial cells

31 March 2020

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

31 March 2020

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

31 March 2020

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

31 March 2020

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

31 March 2020

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

31 March 2020

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Gene therapy for muscle disease

31 March 2020

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

31 March 2020

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

31 March 2020

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

31 March 2020

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Safety and tolerability of putaminal AADC gene therapy for Parkinson disease

31 March 2020

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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

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

31 March 2020

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

31 March 2020

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

31 March 2020

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

31 March 2020

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Hemophilia gene therapy: a Holy Grail found

31 March 2020

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

31 March 2020

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Current status of polymeric gene delivery systems

31 March 2020

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Nonviral gene delivery: principle, limitations, and recent progress

31 March 2020

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

31 March 2020

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Progress and prospects: nuclear import of nonviral vectors

31 March 2020

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Natural polymers for gene delivery and tissue engineering

31 March 2020

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Gene therapy progress and prospects: hydrodynamic gene delivery

31 March 2020

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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

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

31 March 2020

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

31 March 2020

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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

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The phiC31 integrase system for gene therapy

31 March 2020

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Integration specificity of phage phiC31 integrase in the human genome

31 March 2020

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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

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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

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

31 March 2020

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

31 March 2020

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

31 March 2020

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Adenoviral vector-based strategies for cancer therapy

31 March 2020

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

31 March 2020

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

31 March 2020

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Gefitinib or carboplatin-paclitaxel in pulmonary adenocarcinoma

31 March 2020

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

31 March 2020

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

31 March 2020

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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

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

31 March 2020

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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

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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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