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Future Directions for Adrenal Insufficiency: Cellular Transplantation and Genetic Therapies.
- Published in:
- Journal of Clinical Endocrinology & Metabolism, 2023, v. 108, n. 6, p. 1273, doi. 10.1210/clinem/dgac751
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- Article
Ub-ISAP: a streamlined UNIX pipeline for mining unique viral vector integration sites from next generation sequencing data.
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- BMC Bioinformatics, 2017, v. 18, p. 1, doi. 10.1186/s12859-017-1719-4
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- Publication type:
- Article
Differential subcellular localization of CD86 in human PBMC-derived macrophages and DCs, and ultrastructural characterization by immuno-electron microscopy.
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- International Immunology, 2005, v. 17, n. 2, p. 123
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- Publication type:
- Article
Impact of next-generation sequencing error on analysis of barcoded plasmid libraries of known complexity and sequence.
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- Nucleic Acids Research, 2014, v. 42, n. 16, p. e129, doi. 10.1093/nar/gku607
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- Publication type:
- Article
Steroid hormone receptor gene expression in human breast cancer cells: Inverse relationship between oestrogen and glucocorticoid receptor messenger RNA levels.
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- International Journal of Cancer, 1990, v. 46, n. 6, p. 1081, doi. 10.1002/ijc.2910460622
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- Publication type:
- Article
Gene therapy: Progress in childhood disease.
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- Journal of Paediatrics & Child Health, 2012, v. 48, n. 6, p. 466, doi. 10.1111/j.1440-1754.2011.02204.x
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- Publication type:
- Article
A murine model of neurofibromatosis type 1 tibial pseudarthrosis featuring proliferative fibrous tissue and osteoclast-like cells.
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- Journal of Bone & Mineral Research, 2012, v. 27, n. 1, p. 68, doi. 10.1002/jbmr.528
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- Publication type:
- Article
Methylguanine DNA Methyltransferase-Mediated Drug Resistance-Based Selective Enrichment and Engraftment of Transplanted Stem Cells in Skeletal Muscle.
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- Stem Cells, 2009, v. 27, n. 5, p. 1098, doi. 10.1002/stem.28
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- Publication type:
- Article
A genome-wide map of adeno-associated virus-mediated human gene targeting.
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- Nature Structural & Molecular Biology, 2014, v. 21, n. 11, p. 969, doi. 10.1038/nsmb.2895
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- Publication type:
- Article
Germline viral 'fossils' guide in silico reconstruction of a mid-Cenozoic era marsupial adeno-associated virus.
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- Scientific Reports, 2016, p. 28965, doi. 10.1038/srep28965
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- Publication type:
- Article
Harnessing whole human liver ex situ normothermic perfusion for preclinical AAV vector evaluation.
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- Nature Communications, 2024, v. 15, n. 1, p. 1, doi. 10.1038/s41467-024-46194-y
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- Publication type:
- Article
Robust anti-tumor immunity and memory in Rag-1-deficient mice following adoptive transfer of cytokine-primed splenocytes and tumor CD80 expression.
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- Cancer Immunology, Immunotherapy, 2007, v. 56, n. 12, p. 1955, doi. 10.1007/s00262-007-0339-7
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- Publication type:
- Article
Liver-specific adiponectin gene therapy suppresses microglial NLRP3-inflammasome activation for treating Alzheimer's disease.
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- Journal of Neuroinflammation, 2024, v. 21, n. 1, p. 1, doi. 10.1186/s12974-024-03066-y
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- Publication type:
- Article
ACE2 Therapy Using Adeno-associated Viral Vector Inhibits Liver Fibrosis in Mice.
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- Molecular Therapy, 2015, v. 23, n. 9, p. 1434, doi. 10.1038/mt.2015.92
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- Publication type:
- Article
Adeno-associated Virus-mediated Rescue of Neonatal Lethality in Argininosuccinate Synthetase-deficient Mice.
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- Molecular Therapy, 2013, v. 21, n. 10, p. 1823, doi. 10.1038/mt.2013.139
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- Publication type:
- Article
Gene Therapy Researchers' Assessments Of Risks And Perceptions Of Risk Acceptability In Clinical Trials.
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- Molecular Therapy, 2013, v. 21, n. 4, p. 806, doi. 10.1038/mt.2012.230
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- Publication type:
- Article
Liver-specific deletion of miR-181ab1 reduces liver tumour progression via upregulation of CBX7.
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- Cellular & Molecular Life Sciences, 2022, v. 79, n. 8, p. 1, doi. 10.1007/s00018-022-04452-6
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- Publication type:
- Article
Treatment of an infant with X-linked severe combined immunodeficiency (SCID-X1) by gene therapy in Australia.
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- 2005
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- Publication type:
- journal article
Treatment of an infant with X‐linked severe combined immunodeficiency (SCID‐X1) by gene therapy in Australia.
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- Medical Journal of Australia, 2005, v. 182, n. 9, p. 458, doi. 10.5694/j.1326-5377.2005.tb06785.x
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- Publication type:
- Article
Onasemnogene abeparvovec in spinal muscular atrophy: an Australian experience of safety and efficacy.
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- Annals of Clinical & Translational Neurology, 2022, v. 9, n. 3, p. 339, doi. 10.1002/acn3.51519
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- Publication type:
- Article
Performance of Cardiotropic rAAV Vectors Is Dependent on Production Method.
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- Viruses (1999-4915), 2022, v. 14, n. 8, p. 1623, doi. 10.3390/v14081623
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- Publication type:
- Article
HeLa cells cocultured with peripheral blood lymphocytes acquire an immuno-inhibitory phenotype through up-regulation of indoleamine 2,3-dioxygenase activity.
- Published in:
- Immunology, 2002, v. 105, n. 4, p. 478, doi. 10.1046/j.1365-2567.2002.01390.x
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- Publication type:
- Article
Integrating newborn screening for spinal muscular atrophy into health care systems: an Australian pilot programme.
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- Developmental Medicine & Child Neurology, 2022, v. 64, n. 5, p. 625, doi. 10.1111/dmcn.15117
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- Publication type:
- Article
Gene therapy for urea cycle defects: An update from historical perspectives to future prospects.
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- Journal of Inherited Metabolic Disease, 2024, v. 47, n. 1, p. 50, doi. 10.1002/jimd.12609
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- Publication type:
- Article
Systemic AAV8-mediated delivery of a functional copy of muscle glycogen phosphorylase (Pygm) ameliorates disease in a murine model of McArdle disease.
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- Human Molecular Genetics, 2020, v. 29, n. 1, p. 20, doi. 10.1093/hmg/ddz214
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- Publication type:
- Article
Stable transduction of the neonatal mouse liver using a hybrid rAAV/sleeping beauty transposon gene delivery system.
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- Journal of Gene Medicine, 2024, v. 26, n. 8, p. 1, doi. 10.1002/jgm.3726
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- Publication type:
- Article
Gene therapy clinical trials worldwide to 2023—an update.
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- Journal of Gene Medicine, 2024, v. 26, n. 8, p. 1, doi. 10.1002/jgm.3721
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- Publication type:
- Article
Partial pancreatic transdifferentiation of primary human hepatocytes in the livers of a humanised mouse model.
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- Journal of Gene Medicine, 2018, v. 20, n. 5, p. 1, doi. 10.1002/jgm.3017
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- Publication type:
- Article
Gene therapy clinical trials worldwide to 2017: An update.
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- Journal of Gene Medicine, 2018, v. 20, n. 5, p. 1, doi. 10.1002/jgm.3015
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- Publication type:
- Article
Gene therapy clinical trials worldwide to 2012 - an update.
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- Journal of Gene Medicine, 2013, v. 15, n. 2, p. 65, doi. 10.1002/jgm.2698
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- Publication type:
- Article
Developing strategies for detection of gene doping.
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- Journal of Gene Medicine, 2008, v. 10, n. 1, p. 3, doi. 10.1002/jgm.1114
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- Publication type:
- Article
Lentivirus vector-mediated gene transfer to the developing bronchiolar airway epithelium in the fetal lamb.
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- Journal of Gene Medicine, 2007, v. 9, n. 6, p. 429, doi. 10.1002/jgm.1039
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- Publication type:
- Article
Characterisation of a P140K mutant O<sup>6</sup>-methylguanine-DNA-methyltransferase (MGMT)-expressing transgenic mouse line with drug-selectable bone marrow.
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- Journal of Gene Medicine, 2006, v. 8, n. 9, p. 1071, doi. 10.1002/jgm.937
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- Publication type:
- Article
Thymocyte self-renewal and oncogenic risk in immunodeficient mouse models: relevance for human gene therapy clinical trials targeting haematopoietic stem cell populations?
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- Mammalian Genome, 2018, v. 29, n. 11/12, p. 771, doi. 10.1007/s00335-018-9780-5
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- Publication type:
- Article
Selection and evaluation of clinically relevant AAV variants in a xenograft liver model.
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- Nature, 2014, v. 506, n. 7488, p. 382, doi. 10.1038/nature12875
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- Publication type:
- Article
Induction and Prevention of Severe Hyperammonemia in the spf<sup>ash</sup> Mouse Model of Ornithine Transcarbamylase Deficiency Using shRNA and rAAV-mediated Gene Delivery.
- Published in:
- Molecular Therapy, 2011, v. 19, n. 5, p. 854, doi. 10.1038/mt.2011.32
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- Publication type:
- Article
Lymphomagenesis in SCID-X1 Mice Following Lentivirus-mediated Phenotype Correction Independent of Insertional Mutagenesis and γc Overexpression.
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- Molecular Therapy, 2010, v. 18, n. 5, p. 965, doi. 10.1038/mt.2010.50
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- Publication type:
- Article
Accepting Risk in Clinical Research: Is the Gene Therapy Field Becoming Too Risk-averse?
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- Molecular Therapy, 2009, v. 17, n. 11, p. 1842, doi. 10.1038/mt.2009.223
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- Publication type:
- Article
Sexually Dimorphic Patterns of Episomal rAAV Genome Persistence in the Adult Mouse Liver and Correlation With Hepatocellular Proliferation.
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- Molecular Therapy, 2009, v. 17, n. 9, p. 1548, doi. 10.1038/mt.2009.139
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- Publication type:
- Article
AAV2/8-mediated Correction of OTC Deficiency Is Robust in Adult but Not Neonatal Spf<sup>ash</sup> Mice.
- Published in:
- Molecular Therapy, 2009, v. 17, n. 8, p. 1340, doi. 10.1038/mt.2009.88
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- Publication type:
- Article
Gene Delivery to the Juvenile Mouse Liver Using AAV2/8 Vectors.
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- Molecular Therapy, 2008, v. 16, n. 6, p. 1081, doi. 10.1038/mt.2008.72
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- Publication type:
- Article
Potential Use of Gene Transfer in Athletic Performance Enhancement.
- Published in:
- Molecular Therapy, 2007, v. 15, n. 10, p. 1751, doi. 10.1038/sj.mt.6300278
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- Publication type:
- Article
1103. Augmentation of Immunity Against AAV- Encoded Transgenes by Use of the Genetic Adjuvants C3d3 and CTLA4.
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- Molecular Therapy, 2006, v. 13, p. S424, doi. 10.1016/j.ymthe.2006.08.1207
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- Publication type:
- Article
839. Lentiviral Vectors Encoding Connexin43 Mutants Reduce Gap Junction Function: Toward Molecular Ablation of Re-Entrant Cardiac Arrhythmias
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- 2005
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- Publication type:
- Abstract
Use of a Hybrid Adeno-Associated Viral Vector Transposon System to Deliver the Insulin Gene to Diabetic NOD Mice.
- Published in:
- Cells (2073-4409), 2020, v. 9, n. 10, p. 2227, doi. 10.3390/cells9102227
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- Publication type:
- Article
Antigen fusion with C3d<sub>3</sub> augments or inhibits humoral immunity to AAV genetic vaccines in a transgene-dependent manner.
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- Immunology & Cell Biology, 2010, v. 88, n. 2, p. 228, doi. 10.1038/icb.2009.92
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- Publication type:
- Article
Brief Communication CD4 expression on EL4 cells as an epiphenomenon of retroviral transduction and selection.
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- Immunology & Cell Biology, 2004, v. 82, n. 2, p. 132, doi. 10.1046/j.0818-9641.2004.01228.x
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- Publication type:
- Article
Restoring the natural tropism of AAV2 vectors for human liver.
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- Science Translational Medicine, 2020, v. 12, n. 560, p. 1, doi. 10.1126/scitranslmed.aba3312
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- Publication type:
- Article
Myoblast sensitivity and fibroblast insensitivity to osteogenic conversion by BMP-2 correlates with the expression of Bmpr-1a.
- Published in:
- BMC Musculoskeletal Disorders, 2009, v. 10, p. 1, doi. 10.1186/1471-2474-10-51
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- Publication type:
- Article
A novel splice-site mutation in the common gamma chain (γc) gene IL2RG results in X-linked severe combined immunodeficiency with an atypical NK<sup>+</sup> phenotype.
- Published in:
- Human Mutation, 2004, v. 23, n. 5, p. 522, doi. 10.1002/humu.9235
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- Publication type:
- Article