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261st ENMC International Workshop: Management of safety issues arising following AAV gene therapy. 17th-19th June 2022, Hoofddorp, The Netherlands

Neuromuscular disorders : NMD, 2023-11, Vol.33 (11), p.884-896

2023

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A processed pseudogene codes for a new antigen recognized by a CD8(+) T cell clone on melanoma

The Journal of experimental medicine, 2000-05, Vol.191 (9), p.1617-1624

2000

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High Avidity Melanoma-reactive Cytotoxic T Lymphocytes Are Efficiently Induced from Peripheral Blood Lymphocytes on Stimulation by Peptide-pulsed Melanoma Cells

Clinical cancer research, 2000-04, Vol.6 (4), p.1459-1467

2000

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LFA‐3 co‐stimulates cytokine secretion by cytotoxic T lymphocytes by providing a TCR‐independent activation signal

European journal of immunology, 1998-04, Vol.28 (4), p.1322-1331

1998

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Heterogeneity of biologic responses of melanoma-specific CTL

The Journal of immunology (1950), 1997-09, Vol.159 (6), p.2831-2839

1997

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Optimal T cell activation by melanoma cells depends on a minimal level of antigen transcription

The Journal of immunology (1950), 1997-02, Vol.158 (3), p.1238-1245

1997

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A Processed Pseudogene Codes for a New Antigen Recognized by a CD8 super(+) T Cell Clone on Melanoma

The Journal of experimental medicine, 2000-05, Vol.191 (9), p.1617-1624

2000

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Two fathers for twin sisters

International Congress series, 2003, Vol.1239, p.933-937

2003

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Frequency and relative fraction of tumor antigen‐specific T cells among lymphocytes from melanoma‐invaded lymph nodes

International journal of cancer, 1998-10, Vol.78 (2), p.209-215

1998

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Efficient central nervous system AAVrh10-mediated intrathecal gene transfer in adult and neonate rats

Gene therapy, 2015-04, Vol.22 (4), p.316-324

2015

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Establishment of two quantitative nested qPCR assays targeting the human EPO transgene

Gene therapy, 2016-04, Vol.23 (4), p.330-339

2016

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AAV2/9-mediated silencing of PMP22 prevents the development of pathological features in a rat model of Charcot-Marie-Tooth disease 1 A

Nature communications, 2021-04, Vol.12 (1), p.2356-2356, Article 2356

2021

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Characterization of dystrophin deficient rats: a new model for Duchenne muscular dystrophy

PloS one, 2014-10, Vol.9 (10), p.e110371

2014

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Serum Profiling Identifies Novel Muscle miRNA and Cardiomyopathy-Related miRNA Biomarkers in Golden Retriever Muscular Dystrophy Dogs and Duchenne Muscular Dystrophy Patients

The American journal of pathology, 2014-11, Vol.184 (11), p.2885-2898

2014

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Five Years of Successful Inducible Transgene Expression Following Locoregional Adeno-Associated Virus Delivery in Nonhuman Primates with No Detectable Immunity

Human gene therapy, 2019-07, Vol.30 (7), p.802-813

2019

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Longevity of rAAV vector and plasmid DNA in blood after intramuscular injection in nonhuman primates: implications for gene doping

Gene therapy, 2011-07, Vol.18 (7), p.709-718

2011

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Evaluation of the detection of the homologous transfusion of a red blood cell concentrate in vivo for antidoping

Drug testing and analysis, 2023-11, Vol.15 (11-12), p.1417-1429

2023

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Adeno-Associated Virus Vector Genomes Persist as Episomal Chromatin in Primate Muscle

Journal of Virology, 2008-08, Vol.82 (16), p.7875-7885

2008

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Dystrophin and mini-dystrophin quantification by mass spectrometry in skeletal muscle for gene therapy development in Duchenne muscular dystrophy

Gene therapy, 2022-11, Vol.29 (10-11), p.608-615

2022

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TRPC3, but not TRPC1, as a good therapeutic target for standalone or complementary treatment of DMD

Journal of translational medicine, 2021-12, Vol.19 (1), p.519-519, Article 519

2021

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Characterization of brain dystrophins absence and impact in dystrophin-deficient Dmdmdx rat model

PloS one, 2020, Vol.15 (3), p.e0230083-e0230083

2020

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The RNA-Binding Protein TIA-1 Is a Novel Mammalian Splicing Regulator Acting through Intron Sequences Adjacent to a 5′ Splice Site

Molecular and Cellular Biology, 2000-09, Vol.20 (17), p.6287-6299

2000

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Liver-Specific Transcriptional Modules Identified by Genome-Wide In Silico Analysis Enable Efficient Gene Therapy in Mice and Non-Human Primates

Molecular therapy, 2014-09, Vol.22 (9), p.1605-1613

2014

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Novel chemical tyrosine functionalization of adeno-associated virus improves gene transfer efficiency in liver and retina

Biomedicine & pharmacotherapy, 2024-02, Vol.171, p.116148-116148, Article 116148

2024

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261st ENMC International Workshop: Management of safety issues arising following AAV gene therapy. 17th-19th June 2022, Hoofddorp, The Netherlands

A processed pseudogene codes for a new antigen recognized by a CD8(+) T cell clone on melanoma

High Avidity Melanoma-reactive Cytotoxic T Lymphocytes Are Efficiently Induced from Peripheral Blood Lymphocytes on Stimulation by Peptide-pulsed Melanoma Cells

LFA‐3 co‐stimulates cytokine secretion by cytotoxic T lymphocytes by providing a TCR‐independent activation signal

Heterogeneity of biologic responses of melanoma-specific CTL

Optimal T cell activation by melanoma cells depends on a minimal level of antigen transcription

A Processed Pseudogene Codes for a New Antigen Recognized by a CD8 super(+) T Cell Clone on Melanoma

Two fathers for twin sisters

Frequency and relative fraction of tumor antigen‐specific T cells among lymphocytes from melanoma‐invaded lymph nodes

Efficient central nervous system AAVrh10-mediated intrathecal gene transfer in adult and neonate rats

Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy

Establishment of two quantitative nested qPCR assays targeting the human EPO transgene

AAV2/9-mediated silencing of PMP22 prevents the development of pathological features in a rat model of Charcot-Marie-Tooth disease 1 A

Characterization of dystrophin deficient rats: a new model for Duchenne muscular dystrophy

Serum Profiling Identifies Novel Muscle miRNA and Cardiomyopathy-Related miRNA Biomarkers in Golden Retriever Muscular Dystrophy Dogs and Duchenne Muscular Dystrophy Patients

Five Years of Successful Inducible Transgene Expression Following Locoregional Adeno-Associated Virus Delivery in Nonhuman Primates with No Detectable Immunity

Longevity of rAAV vector and plasmid DNA in blood after intramuscular injection in nonhuman primates: implications for gene doping

Evaluation of the detection of the homologous transfusion of a red blood cell concentrate in vivo for antidoping

Adeno-Associated Virus Vector Genomes Persist as Episomal Chromatin in Primate Muscle

Dystrophin and mini-dystrophin quantification by mass spectrometry in skeletal muscle for gene therapy development in Duchenne muscular dystrophy

TRPC3, but not TRPC1, as a good therapeutic target for standalone or complementary treatment of DMD

Characterization of brain dystrophins absence and impact in dystrophin-deficient Dmdmdx rat model

The RNA-Binding Protein TIA-1 Is a Novel Mammalian Splicing Regulator Acting through Intron Sequences Adjacent to a 5′ Splice Site

Liver-Specific Transcriptional Modules Identified by Genome-Wide In Silico Analysis Enable Efficient Gene Therapy in Mice and Non-Human Primates

Novel chemical tyrosine functionalization of adeno-associated virus improves gene transfer efficiency in liver and retina

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