Found: 56
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Systemic gene therapy rescues retinal dysfunction and hearing loss in a model of Norrie disease.
- Published in:
- EMBO Molecular Medicine, 2023, v. 15, n. 10, p. 1, doi. 10.15252/emmm.202317393
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- Publication type:
- Article
Beclin‐1‐mediated activation of autophagy improves proximal and distal urea cycle disorders.
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- EMBO Molecular Medicine, 2021, v. 13, n. 2, p. 1, doi. 10.15252/emmm.202013158
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- Publication type:
- Article
Activation and deactivation of periventricular white matter phagocytes during postnatal mouse development.
- Published in:
- Glia, 2010, v. 58, n. 1, p. 11, doi. 10.1002/glia.20896
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- Publication type:
- Article
Evidence for Contribution of CD4+CD25+ Regulatory T Cells in Maintaining Immune Tolerance to Human Factor IX following Perinatal Adenovirus Vector Delivery.
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- Journal of Immunology Research, 2015, v. 2015, p. 1, doi. 10.1155/2015/397879
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- Publication type:
- Article
Evidence for contribution of CD4+ CD25+ regulatory T cells in maintaining immune tolerance to human factor IX following perinatal adenovirus vector delivery.
- Published in:
- 2015
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- Publication type:
- journal article
Urea Cycle Related Amino Acids Measured in Dried Bloodspots Enable Long-Term In Vivo Monitoring and Therapeutic Adjustment.
- Published in:
- Metabolites (2218-1989), 2019, v. 9, n. 11, p. 275, doi. 10.3390/metabo9110275
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- Publication type:
- Article
Widespread Distribution and Muscle Differentiation of Human Fetal Mesenchymal Stem Cells After Intrauterine Transplantation in Dystrophic mdx Mouse.
- Published in:
- Stem Cells, 2007, v. 25, n. 4, p. 875, doi. 10.1634/stemcells.2006-0694
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- Publication type:
- Article
Erratum: Lentiviral vectors can be used for full-length dystrophin gene therapy.
- Published in:
- Scientific Reports, 2017, p. 46880, doi. 10.1038/srep46880
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- Publication type:
- Article
Lentiviral vectors can be used for full-length dystrophin gene therapy.
- Published in:
- Scientific Reports, 2017, p. 44775, doi. 10.1038/srep44775
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- Publication type:
- Article
Longitudinal in vivo bioimaging of hepatocyte transcription factor activity following cholestatic liver injury in mice.
- Published in:
- Scientific Reports, 2017, p. 41874, doi. 10.1038/srep41874
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- Publication type:
- Article
Regionally-Specified Second Trimester Fetal Neural Stem Cells Reveals Differential Neurogenic Programming.
- Published in:
- PLoS ONE, 2014, v. 9, n. 9, p. 1, doi. 10.1371/journal.pone.0105985
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- Publication type:
- Article
Gene Therapy with Adeno-associated Virus for Cystic Fibrosis.
- Published in:
- 2016
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- Publication type:
- commentary
Development of S/MAR minicircles for enhanced and persistent transgene expression in the mouse liver.
- Published in:
- Journal of Molecular Medicine, 2011, v. 89, n. 5, p. 515, doi. 10.1007/s00109-010-0713-3
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- Publication type:
- Article
Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development.
- Published in:
- Biomolecules (2218-273X), 2021, v. 11, n. 4, p. 611, doi. 10.3390/biom11040611
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- Publication type:
- Article
Transduction of Fetal Mice With a Feline Lentiviral Vector Induces Liver Tumors Which Exhibit an E2F Activation Signature.
- Published in:
- Molecular Therapy, 2014, v. 22, n. 1, p. 59, doi. 10.1038/mt.2013.193
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- Publication type:
- Article
Exon Skipping of Hepatic APOB Pre-mRNA With Splice-switching Oligonucleotides Reduces LDL Cholesterol In Vivo.
- Published in:
- Molecular Therapy, 2013, v. 21, n. 3, p. 602, doi. 10.1038/mt.2012.264
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- Publication type:
- Article
The Fetal Mouse Is a Sensitive Genotoxicity Model That Exposes Lentiviral-associated Mutagenesis Resulting in Liver Oncogenesis.
- Published in:
- Molecular Therapy, 2013, v. 21, n. 2, p. 324, doi. 10.1038/mt.2012.224
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- Publication type:
- Article
Human HPSE2 gene transfer ameliorates bladder pathophysiology in a mutant mouse model of urofacial syndrome.
- Published in:
- eLife, 2024, p. 1, doi. 10.7554/eLife.91828
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- Publication type:
- Article
Generation of light-producing somatic-transgenic mice using adeno-associated virus vectors.
- Published in:
- Scientific Reports, 2020, v. 10, n. 1, p. 1, doi. 10.1038/s41598-020-59075-3
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- Publication type:
- Article
Publisher Correction: In Utero Gene Therapy (IUGT) Using GLOBE Lentiviral Vector Phenotypically Corrects the Heterozygous Humanised Mouse Model and Its Progress Can Be Monitored Using MRI Techniques.
- Published in:
- 2019
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- Publication type:
- Correction Notice
In Utero Gene Therapy (IUGT) Using GLOBE Lentiviral Vector Phenotypically Corrects the Heterozygous Humanised Mouse Model and Its Progress Can Be Monitored Using MRI Techniques.
- Published in:
- Scientific Reports, 2019, v. 9, n. 1, p. N.PAG, doi. 10.1038/s41598-019-48078-4
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- Publication type:
- Article
Therapeutic expression of human clotting factors IX and X following adeno-associated viral vector-mediated intrauterine gene transfer in early-gestation fetal macaques.
- Published in:
- FASEB Journal, 2019, v. 33, n. 3, p. 3954, doi. 10.1096/fj.201801391R
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- Publication type:
- Article
Systemic gene delivery following intravenous administration of AAV9 to fetal and neonatal mice and late-gestation nonhuman primates.
- Published in:
- FASEB Journal, 2015, v. 29, n. 9, p. 3876, doi. 10.1096/fj.14-269092
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- Publication type:
- Article
Activator protein 1 is a key terminal mediator of inflammation-induced preterm labor in mice.
- Published in:
- FASEB Journal, 2014, v. 28, n. 5, p. 2358, doi. 10.1096/fj.13-247783
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- Publication type:
- Article
Intravenous administration of AAV2/9 to the fetal and neonatal mouse leads to differential targeting of CNS cell types and extensive transduction of the nervous system.
- Published in:
- FASEB Journal, 2011, v. 25, n. 10, p. 3505, doi. 10.1096/fj.11-182311
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- Publication type:
- Article
Impaired folate 1-carbon metabolism causes formate-preventable hydrocephalus in glycine decarboxylase-deficient mice.
- Published in:
- 2020
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- Publication type:
- journal article
The CD46-Jagged1 interaction is critical for human T<sub>H</sub>1 immunity.
- Published in:
- Nature Immunology, 2012, v. 13, n. 12, p. 1213, doi. 10.1038/ni.2454
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- Publication type:
- Article
Comparison of different promoters to improve AAV vector-mediated gene therapy for neuronopathic Gaucher disease.
- Published in:
- Human Molecular Genetics, 2024, v. 33, n. 17, p. 1467, doi. 10.1093/hmg/ddae081
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- Publication type:
- Article
In Vitro and In Vivo Evaluation of Human Adenovirus Type 49 as a Vector for Therapeutic Applications.
- Published in:
- Viruses (1999-4915), 2021, v. 13, n. 8, p. 1483, doi. 10.3390/v13081483
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- Publication type:
- Article
Gene therapy for neurotransmitter‐related disorders.
- Published in:
- Journal of Inherited Metabolic Disease, 2024, v. 47, n. 1, p. 176, doi. 10.1002/jimd.12697
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- Publication type:
- Article
Fetal gene therapy.
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- Journal of Inherited Metabolic Disease, 2024, v. 47, n. 1, p. 192, doi. 10.1002/jimd.12659
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- Publication type:
- Article
Fetal gene therapy for neurodegenerative lysosomal storage diseases.
- Published in:
- Journal of Inherited Metabolic Disease, 2019, v. 42, n. 3, p. 391, doi. 10.1002/jimd.12018
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- Publication type:
- Article
Systemic AAV9 gene therapy using the synapsin I promoter rescues a mouse model of neuronopathic Gaucher disease but with limited cross-correction potential to astrocytes.
- Published in:
- Human Molecular Genetics, 2020, v. 29, n. 12, p. 1933, doi. 10.1093/hmg/ddz317
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- Publication type:
- Article
Delivery and long-term expression of a 135 kb LDLR genomic DNA locus in vivo by hydrodynamic tail vein injection.
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- Journal of Gene Medicine, 2007, v. 9, n. 6, p. 488, doi. 10.1002/jgm.1041
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- Publication type:
- Article
Gene Therapy for Dopamine Dyshomeostasis: From Parkinson's to Primary Neurotransmitter Diseases.
- Published in:
- Movement Disorders, 2023, v. 38, n. 6, p. 924, doi. 10.1002/mds.29416
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- Publication type:
- Article
Stable Human FIX Expression After 0.9G Intrauterine Gene Transfer of Self-complementary Adeno-associated Viral Vector 5 and 8 in Macaques.
- Published in:
- Molecular Therapy, 2011, v. 19, n. 11, p. 1950, doi. 10.1038/mt.2011.107
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- Publication type:
- Article
Long-term Safety and Efficacy Following Systemic Administration of a Self-complementary AAV Vector Encoding Human FIX Pseudotyped With Serotype 5 and 8 Capsid Proteins.
- Published in:
- Molecular Therapy, 2011, v. 19, n. 5, p. 876, doi. 10.1038/mt.2010.274
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- Publication type:
- Article
Gene Delivery of a Mutant TGFβ3 Reduces Markers of Scar Tissue Formation After Cutaneous Wounding.
- Published in:
- Molecular Therapy, 2010, v. 18, n. 12, p. 2104, doi. 10.1038/mt.2010.174
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- Publication type:
- Article
Neonatal Gene Therapy of Glycogen Storage Disease Type Ia Using a Feline Immunodeficiency Virus–based Vector.
- Published in:
- Molecular Therapy, 2010, v. 18, n. 9, p. 1592, doi. 10.1038/mt.2010.119
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- Publication type:
- Article
Desmin-regulated Lentiviral Vectors for Skeletal Muscle Gene Transfer.
- Published in:
- Molecular Therapy, 2010, v. 18, n. 3, p. 601, doi. 10.1038/mt.2009.267
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- Publication type:
- Article
Influence of Coagulation Factor X on In Vitro and In Vivo Gene Delivery by Adenovirus (Ad) 5, Ad35, and Chimeric Ad5/Ad35 Vectors.
- Published in:
- Molecular Therapy, 2009, v. 17, n. 10, p. 1683, doi. 10.1038/mt.2009.152
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- Publication type:
- Article
Stable Gene Transfer to Muscle Using Non-integrating Lentiviral Vectors.
- Published in:
- Molecular Therapy, 2007, v. 15, n. 11, p. 1947, doi. 10.1038/sj.mt.6300281
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- Publication type:
- Article
The Influence of Blood on In Vivo Adenovirus Bio-distribution and Transduction.
- Published in:
- Molecular Therapy, 2007, v. 15, n. 8, p. 1410, doi. 10.1038/sj.mt.6300206
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- Publication type:
- Article
376. Hepatic Tropism of Adenoviral Type 5 Vectors Can Be Mediated by Multiple Coagulation Factors.
- Published in:
- Molecular Therapy, 2006, v. 13, p. S143, doi. 10.1016/j.ymthe.2006.08.436
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- Publication type:
- Article
827. Oncogenesis Following Delivery of Lentiviral Vectors to Fetal and Neonatal Mice.
- Published in:
- Molecular Therapy, 2006, v. 13, p. S320, doi. 10.1016/j.ymthe.2006.08.912
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- Publication type:
- Article
Corrigendum to “Oncogenesis Following Delivery of a Nonprimate Lentiviral Gene Therapy Vector to Fetal and Neonatal Mice”.
- Published in:
- Molecular Therapy, 2006, v. 13, n. 4, p. 830, doi. 10.1016/j.ymthe.2005.10.003
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- Publication type:
- Article
Oncogenesis Following Delivery of a Nonprimate Lentiviral Gene Therapy Vector to Fetal and Neonatal Mice
- Published in:
- Molecular Therapy, 2005, v. 12, n. 4, p. 763, doi. 10.1016/j.ymthe.2005.07.358
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- Publication type:
- Article
394. Oncogenesis Following Delivery of a Non-Primate Lentiviral Gene Therapy Vector to Fetal Mice
- Published in:
- 2005
- By:
- Publication type:
- Abstract
238. Stem Cell Gene Delivery to Immunocompetent Mice Using a Non-Myeloablative Regimen Results in Long-Term Transgene Expression and Stable Mixed Chimerism
- Published in:
- 2005
- By:
- Publication type:
- Abstract
In Utero gene therapy: current challenges and perspectives
- Published in:
- Molecular Therapy, 2005, v. 11, n. 5, p. 661, doi. 10.1016/j.ymthe.2005.01.015
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- Publication type:
- Article