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Identification of a myotropic AAV by massively parallel in vivo evaluation of barcoded capsid variants.
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- Nature Communications, 2020, v. 11, n. 1, p. N.PAG, doi. 10.1038/s41467-020-19230-w
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- Article
Genetic and Epigenetic Modification of Rat Liver Progenitor Cells via HNF4α Transduction and 5' Azacytidine Treatment: An Integrated miRNA and mRNA Expression Profile Analysis.
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- Genes, 2020, v. 11, n. 5, p. 486, doi. 10.3390/genes11050486
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- Article
Towards a global multidisciplinary consensus framework on haemophilia gene therapy: Report of the 2nd World Federation of Haemophilia Gene Therapy Round Table.
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- Haemophilia, 2020, v. 26, n. 3, p. 443, doi. 10.1111/hae.13971
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- Article
Preclinical and clinical advances in transposon-based gene therapy.
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- Bioscience Reports, 2017, v. 37, n. 6, p. 1, doi. 10.1042/BSR20160614
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- Article
piggyBac transposons expressing full-length human dystrophin enable genetic correction of dystrophic mesoangioblasts.
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- Nucleic Acids Research, 2016, v. 44, n. 2, p. 744, doi. 10.1093/nar/gkv1464
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- Article
Gene therapy for cardiovascular disease: advances in vector development, targeting, and delivery for clinical translation.
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- Cardiovascular Research, 2015, v. 108, n. 1, p. 4, doi. 10.1093/cvr/cvv205
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- Article
Corrigendum: Efficient derivation and inducible differentiation of expandable skeletal myogenic cells from human ES and patient-specific iPS cells.
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- Nature Protocols, 2015, v. 10, n. 9, p. 1457, doi. 10.1038/nprot0915-1457d
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- Article
Efficient derivation and inducible differentiation of expandable skeletal myogenic cells from human ES and patient-specific iPS cells.
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- Nature Protocols, 2015, v. 10, n. 7, p. 941, doi. 10.1038/nprot.2015.057
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- Article
Moving Forward Toward a Cure for Hemophilia B.
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- Molecular Therapy, 2015, v. 23, n. 5, p. 809, doi. 10.1038/mt.2015.56
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- Article
Liver-directed lentiviral gene therapy in a dog model of hemophilia B.
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- Science Translational Medicine, 2015, v. 7, n. 277, p. 1, doi. 10.1126/scitranslmed.aaa1405
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- Article
Hitting the Target Without Pulling the Trigger.
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- Molecular Therapy, 2015, v. 23, n. 1, p. 4, doi. 10.1038/mt.2014.230
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- Article
Genome-wide Computational Analysis Reveals Cardiomyocyte-specific Transcriptional Cis-regulatory Motifs That Enable Efficient Cardiac Gene Therapy.
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- Molecular Therapy, 2015, v. 23, n. 1, p. 43, doi. 10.1038/mt.2014.178
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- Article
Hyperactive PiggyBac Transposons for Sustained and Robust Liver-targeted Gene Therapy.
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- Molecular Therapy, 2014, v. 22, n. 9, p. 1614, doi. 10.1038/mt.2014.131
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- Article
Vector Decoys Trick the Immune Response.
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- Science Translational Medicine, 2013, v. 5, n. 194, p. 1, doi. 10.1126/scitranslmed.3006633
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- Article
Low hippocampal PI(4,5)P<sub>2</sub> contributes to reduced cognition in old mice as a result of loss of MARCKS.
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- Nature Neuroscience, 2013, v. 16, n. 4, p. 449, doi. 10.1038/nn.3342
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- Article
Repression of Cardiac Hypertrophy by KLF15: Underlying Mechanisms and Therapeutic Implications.
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- PLoS ONE, 2012, v. 7, n. 5, p. 1, doi. 10.1371/journal.pone.0036754
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- Article
Thrombospondin-2 prevents cardiac injury and dysfunction in viral myocarditis through the activation of regulatory T-cells.
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- Cardiovascular Research, 2012, v. 94, n. 1, p. 115, doi. 10.1093/cvr/cvs077
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- Article
Intrinsic cell memory reinforces myogenic commitment of pericyte-derived iPSCs.
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- Journal of Pathology, 2011, v. 223, n. 5, p. 593, doi. 10.1002/path.2845
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- Article
Retroviral Vectors Induce Epigenetic Chromatin Modifications and IL-10 Production in Transduced B Cells via Activation of Toll-like Receptor 2.
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- Molecular Therapy, 2011, v. 19, n. 4, p. 711, doi. 10.1038/mt.2010.275
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- Article
Gene therapy strategies for hemophilia: benefits versus risks.
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- Journal of Gene Medicine, 2010, v. 12, n. 10, p. 797, doi. 10.1002/jgm.1500
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- Article
Novel Hyperactive Transposons for Genetic Modification of Induced Pluripotent and Adult Stem Cells: A Nonviral Paradigm for Coaxed Differentiation.
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- Stem Cells, 2010, v. 28, n. 10, p. 1760, doi. 10.1002/stem.501
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- Article
Comparative Analysis of Transposable Element Vector Systems in Human Cells.
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- Molecular Therapy, 2010, v. 18, n. 6, p. 1200, doi. 10.1038/mt.2010.47
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- Article
Recent Advances in Lentiviral Vector Development and Applications.
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- Molecular Therapy, 2010, v. 18, n. 3, p. 477, doi. 10.1038/mt.2009.319
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- Article
Molecular evolution of a novel hyperactive Sleeping Beauty transposase enables robust stable gene transfer in vertebrates.
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- Nature Genetics, 2009, v. 41, n. 6, p. 753, doi. 10.1038/ng.343
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- Article
Atonal homolog 1 Is a Tumor Suppressor Gene.
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- PLoS Biology, 2009, v. 7, n. 2, p. e1000039, doi. 10.1371/journal.pbio.1000039
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- Article
Angiogenesis Enhances Factor IX Delivery and Persistence from Retrievable Human Bioengineered Muscle Implants.
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- Molecular Therapy, 2006, v. 14, n. 3, p. 442, doi. 10.1016/j.ymthe.2006.03.019
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- Article
24. Widespread and Efficient Gene Delivery to the Heart and Liver Using AAV Serotype 9: Implications for Cardiovascular Disease and Hemophilia.
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- Molecular Therapy, 2006, v. 13, p. S10, doi. 10.1016/j.ymthe.2006.08.036
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- Article
483. Phenotypic Correction of Von Willebrand Disease Type 3 Blood-Derived Endothelial Cells with Lentiviral Vectors Expressing Von Willebrand Factor.
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- Molecular Therapy, 2006, v. 13, p. S187, doi. 10.1016/j.ymthe.2006.08.552
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- Article
1091. Successful Induction of Immune Tolerance to Canine Factor VIII after Lentiviral-Mediated Gene Therapy in Neonatal Hemophilia A Mice.
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- Molecular Therapy, 2006, v. 13, p. S419, doi. 10.1016/j.ymthe.2006.08.1193
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- Article
Gene therapy flexes muscle.
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- Journal of Gene Medicine, 2005, v. 7, n. 9, p. 1255, doi. 10.1002/jgm.814
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- Article
40. Enhanced Factor IX Delivery from Bioengineered Hybrid Human Skeletal Muscle Co-Expressing VEGF
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- 2005
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- Abstract
Nanoparticles for the delivery of genes and drugs to human hepatocytes.
- Published in:
- Nature Biotechnology, 2003, v. 21, n. 8, p. 885, doi. 10.1038/nbt843
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- Article
Therapeutic levels of human Factor VIII in mice implanted with encapsulated cells: potential for gene therapy of haemophilia A.
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- Journal of Gene Medicine, 2002, v. 4, n. 2, p. 215, doi. 10.1002/jgm.248
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- Article
Synergism between vascular endothelial growth factor and placental growth factor contributes to angiogenesis and plasma extravasation in pathological conditions.
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- Nature Medicine, 2001, v. 7, n. 5, p. 575, doi. 10.1038/87904
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- Article
Gene therapy for hemophilia.
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- Journal of Gene Medicine, 2001, v. 3, n. 1, p. 3, doi. 10.1002/1521-2254(200101/02)3:1<3::AID-JGM167>3.0.CO;2-H
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- Article
Suggestive evidence that genes controlling invasion and metastasis of T-cell lymphomas are located on mouse chromosome 3.
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- Genes, Chromosomes & Cancer, 1989, v. 1, n. 2, p. 119, doi. 10.1002/gcc.2870010203
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- Article