Engineering adeno-associated virus vectors for gene therapy (Q89637735)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:32042148%20AND%20SRC:MED&resulttype=core&format=json

7 April 2020

review article

1 reference

title

Engineering adeno-associated virus vectors for gene therapy (English)

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:32042148%20AND%20SRC:MED&resulttype=core&format=json

7 April 2020

Chengwen Li

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:32042148%20AND%20SRC:MED&resulttype=core&format=json

7 April 2020

R Jude Samulski

series ordinal

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:32042148%20AND%20SRC:MED&resulttype=core&format=json

7 April 2020

publication date

10 February 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:32042148%20AND%20SRC:MED&resulttype=core&format=json

7 April 2020

published in

Nature Reviews Genetics

1 reference

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7 April 2020

volume

1 reference

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7 April 2020

issue

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7 April 2020

page(s)

255-272

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:32042148%20AND%20SRC:MED&resulttype=core&format=json

Gene therapy for age-related macular degeneration.

7 April 2020

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Endgame: glybera finally recommended for approval as the first gene therapy drug in the European union

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7 January 2021

Gene therapy for ADA-SCID, the first marketing approval of an ex vivo gene therapy in Europe: paving the road for the next generation of advanced therapy medicinal products

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7 January 2021

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7 January 2021

Recombinant Adeno-Associated Virus Gene Therapy in Light of Luxturna (and Zolgensma and Glybera): Where Are We, and How Did We Get Here?

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7 January 2021

Adeno-associated virus serotypes: vector toolkit for human gene therapy

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7 January 2021

Adeno-associated virus at 50: a golden anniversary of discovery, research, and gene therapy success--a personal perspective

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7 January 2021

The atomic structure of adeno-associated virus (AAV-2), a vector for human gene therapy.

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7 January 2021

Structurally mapping the diverse phenotype of adeno-associated virus serotype 4.

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7 January 2021

Engineering the AAV capsid to optimize vector-host-interactions

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7 January 2021

Engineering adeno-associated viruses for clinical gene therapy

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7 January 2021

Host determinants of adeno-associated viral vector entry.

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7 January 2021

Mutational analysis of narrow pores at the fivefold symmetry axes of adeno-associated virus type 2 capsids reveals a dual role in genome packaging and activation of phospholipase A2 activity

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7 January 2021

A conformational change in the adeno-associated virus type 2 capsid leads to the exposure of hidden VP1 N termini

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7 January 2021

Novel transcriptional regulatory signals in the adeno-associated virus terminal repeat A/D junction element

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7 January 2021

Optimization of self-complementary AAV vectors for liver-directed expression results in sustained correction of hemophilia B at low vector dose

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7 January 2021

Dystrophin immunity in Duchenne's muscular dystrophy.

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7 January 2021

Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant.

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7 January 2021

Pharmaceutical Development of AAV-Based Gene Therapy Products for the Eye

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7 January 2021

Long-term safety and efficacy of factor IX gene therapy in hemophilia B.

1 reference

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7 January 2021

Adenovirus-associated virus vector-mediated gene transfer in hemophilia B

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

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

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7 January 2021

AAV5-Factor VIII Gene Transfer in Severe Hemophilia A.

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7 January 2021

Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver

1 reference

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7 January 2021

Kinetics of adeno-associated virus serotype 2 (AAV2) and AAV8 capsid antigen presentation in vivo are identical

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7 January 2021

Double-stranded RNA innate immune response activation from long-term adeno-associated virus vector transduction

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7 January 2021

Gene therapy for neurological disorders: progress and prospects

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7 January 2021

Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo

1 reference

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7 January 2021

Self-complementary adeno-associated virus serotype 2 vector: global distribution and broad dispersion of AAV-mediated transgene expression in mouse brain

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Characterization of Adeno-Associated Viral Vector-Mediated Human Factor VIII Gene Therapy in Hemophilia A Mice

1 reference

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7 January 2021

Optimization of Recombinant Adeno-Associated Virus-Mediated Expression for Large Transgenes, Using a Synthetic Promoter and Tandem Array Enhancers

1 reference

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7 January 2021

Liver-specific transcriptional modules identified by genome-wide in silico analysis enable efficient gene therapy in mice and non-human primates

1 reference

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7 January 2021

Genome-wide computational analysis reveals cardiomyocyte-specific transcriptional Cis-regulatory motifs that enable efficient cardiac gene therapy

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7 January 2021

Quantitative adaptation of isoacceptor tRNAs to mRNA codons of alanine, glycine and serine

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7 January 2021

Co-variation of tRNA abundance and codon usage in Escherichia coli at different growth rates.

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7 January 2021

Tissue-specific differences in human transfer RNA expression

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7 January 2021

Target-Cell-Directed Bioengineering Approaches for Gene Therapy of Hemophilia A.

1 reference

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7 January 2021

Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector

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7 January 2021

Expanding AAV packaging capacity with trans-splicing or overlapping vectors: a quantitative comparison

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7 January 2021

Delivering Transgenic DNA Exceeding the Carrying Capacity of AAV Vectors

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7 January 2021

Triple trans-splicing adeno-associated virus vectors capable of transferring the coding sequence for full-length dystrophin protein into dystrophic mice

1 reference

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7 January 2021

Oversized AAV transductifon is mediated via a DNA-PKcs-independent, Rad51C-dependent repair pathway

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7 January 2021

A hybrid vector system expands adeno-associated viral vector packaging capacity in a transgene-independent manner

1 reference

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7 January 2021

Novel Chimeric Gene Therapy Vectors Based on Adeno-Associated Virus and Four Different Mammalian Bocaviruses

1 reference

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7 January 2021

Construction of a recombinant human parvovirus B19: adeno-associated virus 2 (AAV) DNA inverted terminal repeats are functional in an AAV-B19 hybrid virus

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Treatment of a metabolic liver disease by in vivo genome base editing in adult mice

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Intein-mediated protein trans-splicing expands adeno-associated virus transfer capacity in the retina

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

High-Throughput Dissection of AAV-Host Interactions: The Fast and the Curious.

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Engraftment of a galactose receptor footprint onto adeno-associated viral capsids improves transduction efficiency

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

A Rationally Engineered Capsid Variant of AAV9 for Systemic CNS-Directed and Peripheral Tissue-Detargeted Gene Delivery in Neonates.

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

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

1 reference

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7 January 2021

High-efficiency transduction of the mouse retina by tyrosine-mutant AAV serotype vectors

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

High-efficiency transduction and correction of murine hemophilia B using AAV2 vectors devoid of multiple surface-exposed tyrosines.

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Development of Optimized AAV Serotype Vectors for High-Efficiency Transduction at Further Reduced Doses

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Targeted gene delivery to vascular tissue in vivo by tropism-modified adeno-associated virus vectors

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Incorporation of tumor-targeting peptides into recombinant adeno-associated virus capsids

1 reference

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7 January 2021

A NEW RECOMBINANT ADENO-ASSOCIATED VIRUS (AAV)-BASED RANDOM PEPTIDE DISPLAY LIBRARY SYSTEM: INFECTION-DEFECTIVE AAV1.9-3 AS A NOVEL DETARGETED PLATFORM FOR VECTOR EVOLUTION.

1 reference

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7 January 2021

Random peptide libraries displayed on adeno-associated virus to select for targeted gene therapy vectors

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7 January 2021

Pulmonary Targeting of Adeno-associated Viral Vectors by Next-generation Sequencing-guided Screening of Random Capsid Displayed Peptide Libraries

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7 January 2021

Novel random peptide libraries displayed on AAV serotype 9 for selection of endothelial cell-directed gene transfer vectors

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7 January 2021

A brain microvasculature endothelial cell-specific viral vector with the potential to treat neurovascular and neurological diseases.

1 reference

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7 January 2021

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

1 reference

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7 January 2021

Engineering Liver-detargeted AAV9 Vectors for Cardiac and Musculoskeletal Gene Transfer

1 reference

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7 January 2021

In Vivo Selection of a Computationally Designed SCHEMA AAV Library Yields a Novel Variant for Infection of Adult Neural Stem Cells in the SVZ.

1 reference

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7 January 2021

Vector design Tour de Force: integrating combinatorial and rational approaches to derive novel adeno-associated virus variants

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7 January 2021

Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain

1 reference

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7 January 2021

Engineered AAVs for efficient noninvasive gene delivery to the central and peripheral nervous systems

1 reference

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7 January 2021

In vivo-directed evolution of a new adeno-associated virus for therapeutic outer retinal gene delivery from the vitreous

1 reference

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7 January 2021

AAV ancestral reconstruction library enables selection of broadly infectious viral variants.

1 reference

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7 January 2021

In Silico Reconstruction of the Viral Evolutionary Lineage Yields a Potent Gene Therapy Vector

1 reference

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7 January 2021

A synthetic AAV vector enables safe and efficient gene transfer to the mammalian inner ear

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Gene therapy restores auditory and vestibular function in a mouse model of Usher syndrome type 1c.

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Cross-dressing the virion: the transcapsidation of adeno-associated virus serotypes functionally defines subgroups

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Application of polyploid adeno-associated virus vectors for transduction enhancement and neutralizing antibody evasion

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Proteasome inhibitors enhance gene delivery by AAV virus vectors expressing large genomes in hemophilia mouse and dog models: a strategy for broad clinical application

1 reference

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7 January 2021

Mechanistic insights into the enhancement of adeno-associated virus transduction by proteasome inhibitors.

1 reference

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7 January 2021

Arsenic trioxide stabilizes accumulations of adeno-associated virus virions at the perinuclear region, increasing transduction in vitro and in vivo.

1 reference

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7 January 2021

Identification and Validation of Small Molecules That Enhance Recombinant Adeno-associated Virus Transduction following High-Throughput Screens

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7 January 2021

Chemical Modulation of Endocytic Sorting Augments Adeno-associated Viral Transduction

1 reference

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7 January 2021

High-throughput screening identifies kinase inhibitors that increase dual AAV vectors transduction <i>in vitro</i> and in mouse retina.

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Enhancement of Adeno-Associated Virus-Mediated Gene Therapy Using Hydroxychloroquine in Murine and Human Tissues

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Human galectin 3 binding protein interacts with recombinant adeno-associated virus type 6

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

C-reactive protein (CRP) is essential for efficient systemic transduction of recombinant adeno-associated virus vector 1 (rAAV-1) and rAAV-6 in mice

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Direct interaction of human serum proteins with AAV virions to enhance AAV transduction: immediate impact on clinical applications

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

AAV8 virions hijack serum proteins to increase hepatocyte binding for transduction enhancement

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Cryoprecipitate augments the global transduction of the adeno-associated virus serotype 9 after a systemic administration

1 reference

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7 January 2021

AAV-8 and AAV-9 Vectors Cooperate with Serum Proteins Differently Than AAV-1 and AAV-6

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Influence of Pre-existing Anti-capsid Neutralizing and Binding Antibodies on AAV Vector Transduction.

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Systemic gene delivery in large species for targeting spinal cord, brain, and peripheral tissues for pediatric disorders

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Enhancing gene delivery of adeno-associated viruses by cell-permeable peptides

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Blood-brain barrier shuttle peptides enhance AAV transduction in the brain after systemic administration

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Cellular immune response to cryptic epitopes during therapeutic gene transfer

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

An Observational Study from Long-Term AAV Re-administration in Two Hemophilia Dogs

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Gene Delivery of Activated Factor VII Using Alternative Adeno-Associated Virus Serotype Improves Hemostasis in Hemophiliac Mice with FVIII Inhibitors and Adeno-Associated Virus Neutralizing Antibodies

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

The ER-luminal domain of the HCMV glycoprotein US6 inhibits peptide translocation by TAP

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Molecular mechanism and species specificity of TAP inhibition by herpes simplex virus ICP47

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Inhibition of antigen presentation during AAV gene therapy using virus peptides

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Circumventing cellular immunity by miR142-mediated regulation sufficiently supports rAAV-delivered OVA expression without activating humoral immunity.

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Improved induction of immune tolerance to factor IX by hepatic AAV-8 gene transfer

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Adeno-associated virus capsid structure drives CD4-dependent CD8+ T cell response to vector encoded proteins

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

The TLR9-MyD88 pathway is critical for adaptive immune responses to adeno-associated virus gene therapy vectors in mice

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

The genome of self-complementary adeno-associated viral vectors increases Toll-like receptor 9–dependent innate immune responses in the liver

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

CpG-depleted adeno-associated virus vectors evade immune detection.

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

AAV8 Can Induce Innate and Adaptive Immune Response in the Primate Eye.

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Pre-existing anti-adeno-associated virus antibodies as a challenge in AAV gene therapy

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Unraveling the complex story of immune responses to AAV vectors trial after trial

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Structure of adeno-associated virus-2 in complex with neutralizing monoclonal antibody A20

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Adeno-associated virus serotype 1 (AAV1)- and AAV5-antibody complex structures reveal evolutionary commonalities in parvovirus antigenic reactivity

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Single amino acid modification of adeno-associated virus capsid changes transduction and humoral immune profiles

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

High resolution structural characterization of a new AAV5 antibody epitope toward engineering antibody resistant recombinant gene delivery vectors

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Creating an arsenal of Adeno-associated virus (AAV) gene delivery stealth vehicles.

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Structure-guided evolution of antigenically distinct adeno-associated virus variants for immune evasion

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Isolation of targeted AAV2 vectors from novel virus display libraries

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Development of Patient-specific AAV Vectors After Neutralizing Antibody Selection for Enhanced Muscle Gene Transfer

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https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity.

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Adeno-associated virus capsid antigen presentation is dependent on endosomal escape

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Efficient Capsid Antigen Presentation From Adeno-Associated Virus Empty Virions In Vivo.

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Targeted modifications in adeno-associated virus serotype 8 capsid improves its hepatic gene transfer efficiency in vivo

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8+ T cells.

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

How will the field of gene therapy survive its success?

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Large-scale recombinant adeno-associated virus production

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Production of Recombinant Adeno-associated Virus Vectors Using Suspension HEK293 Cells and Continuous Harvest of Vector From the Culture Media for GMP FIX and FLT1 Clinical Vector

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Pharmacology of Recombinant Adeno-associated Virus Production.

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Manufacturing Clinical Grade Recombinant Adeno-Associated Virus Using Invertebrate Cell Lines

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

OneBac: platform for scalable and high-titer production of adeno-associated virus serotype 1-12 vectors for gene therapy

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Direct Head-to-Head Evaluation of Recombinant Adeno-associated Viral Vectors Manufactured in Human versus Insect Cells

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Optimization of viral protein ratios for production of rAAV serotype 5 in the baculovirus system

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

A Robust System for Production of Superabundant VP1 Recombinant AAV Vectors.

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Development of a Novel Recombinant Adeno-Associated Virus Production System Using Human Bocavirus 1 Helper Genes

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Human Bocavirus 1 Is a Novel Helper for Adeno-Associated Virus Replication.

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

A single-step affinity column for purification of serotype-5 based adeno-associated viral vectors.

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Novel tools for production and purification of recombinant adenoassociated virus vectors

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

A simplified baculovirus-AAV expression vector system coupled with one-step affinity purification yields high-titer rAAV stocks from insect cells

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Metabolic biotinylation provides a unique platform for the purification and targeting of multiple AAV vector serotypes

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Engineering adeno-associated virus for one-step purification via immobilized metal affinity chromatography

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Identification of an adeno-associated virus binding epitope for AVB sepharose affinity resin

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Separation of adeno-associated virus type 2 empty particles from genome containing vectors by anion-exchange column chromatography

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Overcoming preexisting humoral immunity to AAV using capsid decoys

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Universal Method for the Purification of Recombinant AAV Vectors of Differing Serotypes

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Intravenous administration of the adeno-associated virus-PHP.B capsid fails to upregulate transduction efficiency in the marmoset brain.

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

The Neurotropic Properties of AAV-PHP.B Are Limited to C57BL/6J Mice.

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Evaluation of engineered AAV capsids for hepatic factor IX gene transfer in murine and canine models

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Systemic administration of AAV8-α-galactosidase A induces humoral tolerance in nonhuman primates despite low hepatic expression

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Selection and evaluation of clinically relevant AAV variants in a xenograft liver model

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Efficient and Targeted Transduction of Nonhuman Primate Liver With Systemically Delivered Optimized AAV3B Vectors.

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Comparative Study of Liver Gene Transfer With AAV Vectors Based on Natural and Engineered AAV Capsids.

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Superior In vivo Transduction of Human Hepatocytes Using Engineered AAV3 Capsid

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Superior human hepatocyte transduction with adeno-associated virus vector serotype 7

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Selecting the Best AAV Capsid for Human Studies

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Human skeletal muscle xenograft as a new preclinical model for muscle disorders

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Humanized Mouse Models of Clinical Disease

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Progress and potential in organoid research

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Photoreceptor precursors derived from three-dimensional embryonic stem cell cultures integrate and mature within adult degenerate retina.

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Stem Cell-Derived Photoreceptor Transplants Differentially Integrate Into Mouse Models of Cone-Rod Dystrophy.

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Complement is an essential component of the immune response to adeno-associated virus vectors

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Gene therapy for inherited retinal and optic nerve degenerations.

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Long-term effect of gene therapy on Leber's congenital amaurosis

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Improvement and decline in vision with gene therapy in childhood blindness.

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Update on clinical gene therapy for hemophilia

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Gene therapy with adeno-associated virus vector 5-human factor IX in adults with hemophilia B.

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Gene therapy for neurological disorders: progress and prospects

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Efficacy and safety of AAV2 gene therapy in children with aromatic L-amino acid decarboxylase deficiency: an open-label, phase 1/2 trial

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Long-term follow-up after gene therapy for canavan disease.

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Systemic AAV Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

The progress of AAV-mediated gene therapy in neuromuscular disorders

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Human Cardiac Gene Therapy

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Calcium upregulation by percutaneous administration of gene therapy in patients with cardiac disease (CUPID 2): a randomised, multinational, double-blind, placebo-controlled, phase 2b trial

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

5 Year Expression and Neutrophil Defect Repair after Gene Therapy in Alpha-1 Antitrypsin Deficiency.

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Phase I open label liver-directed gene therapy clinical trial for acute intermittent porphyria

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Adenovirus-Mediated Gene Delivery: Potential Applications for Gene and Cell-Based Therapies in the New Era of Personalized Medicine

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Adp53 (Gendicine) 12 Years in Clinic - The First Approved Gene Therapy Product for Cancer

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Evolutionary basis of a new gene- and immune-therapeutic approach for the treatment of malignant brain tumors: from mice to clinical trials for glioma patients

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Gene Therapy for "Bubble Boy" Disease

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

First approval in sight for Novartis' CAR-T therapy after panel vote

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

Translating anti-CD19 CAR T-cell therapy into clinical practice for relapsed/refractory diffuse large B-cell lymphoma

1 reference

https://api.crossref.org/works/10.1038%2FS41576-019-0205-4

7 January 2021

The recognition and isolation from urine and serum of a peptide inhibitor to calcification

1 reference

12 December 2020

The postoperative classification for uterine cervical cancer and its clinical evaluation

1 reference

12 December 2020

Protein building blocks preserved by recombination

1 reference

12 December 2020

Evading the immune response upon in vivo gene therapy with viral vectors.

1 reference

12 December 2020

Bioinformatics Analysis of Differential Innate Immune Signaling in Macrophages by Wild-Type Vaccinia Mature Virus and a Mutant Virus with a Deletion of the A26 Protein.

1 reference

12 December 2020

10.1038/S41576-019-0205-4

Identifiers

DOI

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:32042148%20AND%20SRC:MED&resulttype=core&format=json

7 April 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:32042148%20AND%20SRC:MED&resulttype=core&format=json