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Identification of a myotropic AAV by massively parallel in vivo evaluation of barcoded capsid variants

Nature communications, 2020-10, Vol.11 (1), p.5432-5432, Article 5432

2020

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The risk of lower gastrointestinal bleeding in low‐dose aspirin users

Alimentary pharmacology & therapeutics, 2017-06, Vol.45 (12), p.1542-1550

2017

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Molecular evolution of a novel hyperactive Sleeping Beauty transposase enables robust stable gene transfer in vertebrates

Nature genetics, 2009-06, Vol.41 (6), p.753-761

2009

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Transposons: Moving Forward from Preclinical Studies to Clinical Trials

Human gene therapy, 2017-11, Vol.28 (11), p.1087-1104

2017

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Preclinical and clinical advances in transposon-based gene therapy

Bioscience reports, 2017-12, Vol.37 (6)

2017

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Hepatocyte‐targeted expression by integrase‐defective lentiviral vectors induces antigen‐specific tolerance in mice with low genotoxic risk

Hepatology (Baltimore, Md.), 2011-05, Vol.53 (5), p.1696-1707

2011

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Codon optimization of human factor VIII cDNAs leads to high-level expression

Blood, 2011-01, Vol.117 (3), p.798-807

2011

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Hemophilia Gene Therapy: Ready for Prime Time?

Human gene therapy, 2017-11, Vol.28 (11), p.1013-1023

2017

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Type II EATL (epitheliotropic intestinal T-cell lymphoma): a neoplasm of intra-epithelial T-cells with predominant CD8αα phenotype

Leukemia, 2013-08, Vol.27 (8), p.1688-1696

2013

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Recent Advances in Lentiviral Vector Development and Applications

Molecular therapy, 2010-03, Vol.18 (3), p.477-490

2010

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Emerging potential of transposons for gene therapy and generation of induced pluripotent stem cells

Blood, 2009-08, Vol.114 (8), p.1461-1468

2009

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Determination of the small-field output factor for 6 MV photon beam using EGSnrc Monte Carlo

Journal of medical physics, 2022-07, Vol.47 (3), p.301-308

2022

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Efficient CRISPR/Cas9-mediated editing of trinucleotide repeat expansion in myotonic dystrophy patient-derived iPS and myogenic cells

Nucleic acids research, 2018-09, Vol.46 (16), p.8275-8298

2018

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Efficient derivation and inducible differentiation of expandable skeletal myogenic cells from human ES and patient-specific iPS cells

Nature protocols, 2015-07, Vol.10 (7), p.941-958

2015

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JAK-STAT and G-protein-coupled receptor signaling pathways are frequently altered in epitheliotropic intestinal T-cell lymphoma

Leukemia, 2016-06, Vol.30 (6), p.1311-1319

2016

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Gene therapy for hemophilia

Journal of thrombosis and haemostasis, 2013-06, Vol.11, p.99-110

2013

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Hyperactive PiggyBac Transposons for Sustained and Robust Liver-targeted Gene Therapy

Molecular therapy, 2014-09, Vol.22 (9), p.1614-1624

2014

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Liver-directed lentiviral gene therapy in a dog model of hemophilia B

Science translational medicine, 2015-03, Vol.7 (277), p.277ra28

2015

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Distinct transduction of muscle tissue in mice after systemic delivery of AAVpo1 vectors

Gene therapy, 2020-04, Vol.27 (3-4), p.170-179

2020

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Intrinsic cell memory reinforces myogenic commitment of pericyte-derived iPSCs

The Journal of pathology, 2011-04, Vol.223 (5), p.593-603

2011

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Treatment of phenylketonuria using minicircle‐based naked‐DNA gene transfer to murine liver

Hepatology (Baltimore, Md.), 2014-09, Vol.60 (3), p.1035-1043

2014

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MR Imaging Criteria for the Detection of Nasopharyngeal Carcinoma: Discrimination of Early-Stage Primary Tumors from Benign Hyperplasia

American journal of neuroradiology : AJNR, 2018-03, Vol.39 (3), p.515-523

2018

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Computationally designed liver-specific transcriptional modules and hyperactive factor IX improve hepatic gene therapy

Blood, 2014-05, Vol.123 (20), p.3195-3199

2014

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Next-generation muscle-directed gene therapy by in silico vector design

Nature communications, 2019-01, Vol.10 (1), p.492-492, Article 492

2019

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Evaluation of Design Alternatives' Environmental Performance Using AHP and ER Approaches

IEEE systems journal, 2014-12, Vol.8 (4), p.1185-1192

2014

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Identification of a myotropic AAV by massively parallel in vivo evaluation of barcoded capsid variants

The risk of lower gastrointestinal bleeding in low‐dose aspirin users

Molecular evolution of a novel hyperactive Sleeping Beauty transposase enables robust stable gene transfer in vertebrates

Transposons: Moving Forward from Preclinical Studies to Clinical Trials

Preclinical and clinical advances in transposon-based gene therapy

Hepatocyte‐targeted expression by integrase‐defective lentiviral vectors induces antigen‐specific tolerance in mice with low genotoxic risk

Codon optimization of human factor VIII cDNAs leads to high-level expression

Hemophilia Gene Therapy: Ready for Prime Time?

Type II EATL (epitheliotropic intestinal T-cell lymphoma): a neoplasm of intra-epithelial T-cells with predominant CD8αα phenotype

Recent Advances in Lentiviral Vector Development and Applications

Emerging potential of transposons for gene therapy and generation of induced pluripotent stem cells

Determination of the small-field output factor for 6 MV photon beam using EGSnrc Monte Carlo

Efficient CRISPR/Cas9-mediated editing of trinucleotide repeat expansion in myotonic dystrophy patient-derived iPS and myogenic cells

Efficient derivation and inducible differentiation of expandable skeletal myogenic cells from human ES and patient-specific iPS cells

JAK-STAT and G-protein-coupled receptor signaling pathways are frequently altered in epitheliotropic intestinal T-cell lymphoma

Gene therapy for hemophilia

Hyperactive PiggyBac Transposons for Sustained and Robust Liver-targeted Gene Therapy

Liver-directed lentiviral gene therapy in a dog model of hemophilia B

Distinct transduction of muscle tissue in mice after systemic delivery of AAVpo1 vectors

Intrinsic cell memory reinforces myogenic commitment of pericyte-derived iPSCs

Treatment of phenylketonuria using minicircle‐based naked‐DNA gene transfer to murine liver

MR Imaging Criteria for the Detection of Nasopharyngeal Carcinoma: Discrimination of Early-Stage Primary Tumors from Benign Hyperplasia

Computationally designed liver-specific transcriptional modules and hyperactive factor IX improve hepatic gene therapy

Next-generation muscle-directed gene therapy by in silico vector design

Evaluation of Design Alternatives' Environmental Performance Using AHP and ER Approaches

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