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1. Full Text Fetal gene therapy for neurodegenerative lysosomal storage diseases
by Baruteau, Julien and Waddington, Simon N
Journal of inherited metabolic disease, ISSN 0141-8955, 05/2019, Volume 42, Issue 3, pp. 391 - 393
Endocrinology & Metabolism | Genetics & Heredity | Life Sciences & Biomedicine | Medicine, Research & Experimental | Science & Technology | Research & Experimental Medicine | Gene therapy | Lysosomal storage diseases | Fetuses | Index Medicus
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2. Full Text Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects
by Baruteau, Julien and Waddington, Simon N and Alexander, Ian E and Gissen, Paul
Journal of inherited metabolic disease, ISSN 0141-8955, 7/2017, Volume 40, Issue 4, pp. 497 - 517
Human Genetics | Biochemistry, general | Pediatrics | Internal Medicine | Medicine & Public Health | Metabolic Diseases | Hemophilia B - therapy | Dependovirus - genetics | Gene Transfer Techniques | Genetic Therapy | Liver Diseases - genetics | Adenoviridae | Humans | Liver - metabolism | Liver Diseases - therapy | Retroviridae | Treatment Outcome | Hemophilia B - genetics | Hemophilia A - therapy | Animals | Immune System | Mutagenesis | Factor IX - metabolism | Hemophilia A - genetics | Genetic Vectors | Transgenes | DNA, Complementary - metabolism | Antiviral agents | Medical research | Care and treatment | Liver diseases | Children's hospitals | Genes | Respiratory agents | Genetic translation | Prothrombin complex concentrate | Hemophilia | Resveratrol | Medicine, Experimental | Gene therapy | Coagulation factors | Translation | Gene transfer | Therapeutic applications | Risk assessment | Clinical trials | Metabolism | Vectors (Biology) | Expression vectors | Index Medicus | Ssiem 2016
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3. Full Text Mitochondria and Quality Control Defects in a Mouse Model of Gaucher Disease—Links to Parkinson’s Disease
by Osellame, Laura D and Rahim, Ahad A and Hargreaves, Iain P and Gegg, Matthew E and Richard-Londt, Angela and Brandner, Sebastian and Waddington, Simon N and Schapira, Anthony H.V and Duchen, Michael R
Cell metabolism, ISSN 1550-4131, 06/2013, Volume 17, Issue 6, pp. 941 - 953
Endocrinology & Metabolism | Life Sciences & Biomedicine | Science & Technology | Cell Biology | Astrocytes - cytology | Mitochondrial Diseases - genetics | Electron Transport | Glucosylceramidase - genetics | Gaucher Disease - metabolism | Sequestosome-1 Protein | Heat-Shock Proteins - metabolism | Humans | Lysosomes - genetics | Cells, Cultured | Neurons - cytology | Mitochondria - metabolism | Parkinson Disease - genetics | Mice, Knockout | Animals | Gaucher Disease - genetics | Lysosomes - metabolism | Mitochondria - genetics | Mice | Parkinson Disease - metabolism | Autophagy - genetics | Adaptor Proteins, Signal Transducing - metabolism | alpha-Synuclein - metabolism | Disease Models, Animal | Proteins | High technology industry | Neurosciences | Developmental biology | Analysis | Quality control | Mitochondrial DNA | Index Medicus
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4. Full Text NRF2 Orchestrates the Metabolic Shift during Induced Pluripotent Stem Cell Reprogramming
by Hawkins, Kate E and Joy, Shona and Delhove, Juliette M.K.M and Kotiadis, Vassilios N and Fernandez, Emilio and Fitzpatrick, Lorna M and Whiteford, James R and King, Peter J and Bolanos, Juan P and Duchen, Michael R and Waddington, Simon N and McKay, Tristan R
Cell reports (Cambridge), ISSN 2211-1247, 03/2016, Volume 14, Issue 8, pp. 1883 - 1891
Life Sciences & Biomedicine | Science & Technology | Cell Biology | Luciferases - metabolism | Reactive Oxygen Species - metabolism | Transcription Factor AP-1 - genetics | Humans | NF-kappa B - metabolism | Transcription Factor AP-1 - metabolism | Cellular Reprogramming | Luciferases - genetics | Glycolysis - genetics | Kelch-Like ECH-Associated Protein 1 - genetics | Lentivirus - metabolism | Hypoxia-Inducible Factor 1, alpha Subunit - metabolism | Lentivirus - genetics | NF-E2-Related Factor 2 - genetics | Dermis - cytology | Induced Pluripotent Stem Cells - cytology | Genes, Reporter | Fibroblasts - metabolism | Induced Pluripotent Stem Cells - metabolism | Dermis - metabolism | Transduction, Genetic | Genetic Vectors - chemistry | Signal Transduction | Hypoxia-Inducible Factor 1, alpha Subunit - genetics | Gene Expression Regulation | Oxidative Phosphorylation | Genetic Vectors - metabolism | Kelch-Like ECH-Associated Protein 1 - metabolism | NF-kappa B - genetics | NF-E2-Related Factor 2 - metabolism | Fibroblasts - cytology | Pentose Phosphate Pathway - genetics | Index Medicus
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5. Full Text Argininosuccinic aciduria fosters neuronal nitrosative stress reversed by Asl gene transfer
by Baruteau, Julien and Perocheau, Dany P and Hanley, Joanna and Lorvellec, Maëlle and Rocha-Ferreira, Eridan and Karda, Rajvinder and Ng, Joanne and Suff, Natalie and Diaz, Juan Antinao and Rahim, Ahad A and Hughes, Michael P and Banushi, Blerida and Prunty, Helen and Hristova, Mariya and Ridout, Deborah A and Virasami, Alex and Heales, Simon and Howe, Stewen J and Buckley, Suzanne M. K and Mills, Philippa B and Gissen, Paul and Waddington, Simon N
Nature communications, ISSN 2041-1723, 12/2018, Volume 9, Issue 1, pp. 3505 - 14
Science & Technology - Other Topics | Multidisciplinary Sciences | Science & Technology | Brain Diseases - metabolism | Genetic Therapy | Hyperammonemia - genetics | Argininosuccinic Aciduria - metabolism | Argininosuccinate Lyase - genetics | Hyperammonemia - metabolism | Nitrosative Stress - physiology | Brain Diseases - genetics | Citrulline - metabolism | Hyperammonemia - therapy | Argininosuccinic Aciduria - therapy | Nitrosative Stress - genetics | Argininosuccinate Lyase - metabolism | Animals | Argininosuccinic Aciduria - genetics | Brain Diseases - therapy | Liver - cytology | Mice | Neurons - metabolism | Nitric Oxide - metabolism | Stresses | Neonates | Brain | Argininosuccinic aciduria | Intravenous administration | Disease | Gene transfer | Liver | Medical treatment | Cortex | Aciduria | Cognition | Citrulline | Metabolism | ASL gene | Stress | Argininosuccinate lyase | Urea | Ammonia | Cell death | Nitric oxide | Ureas | Metabolic pathways | Metabolic disorders | Index Medicus
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6. Full Text Hyperactive PiggyBac Transposons for Sustained and Robust Liver-targeted Gene Therapy
by Di Matteo, Mario and Samara-Kuko, Emira and Ward, Natalie J and Waddingon, Simon N and McVey, John H and Chuah, Marinee KL and VandenDriessche, Thierry
Molecular therapy, ISSN 1525-0016, 09/2014, Volume 22, Issue 9, pp. 1614 - 1624
Genetics & Heredity | Life Sciences & Biomedicine | Biotechnology & Applied Microbiology | Medicine, Research & Experimental | Science & Technology | Research & Experimental Medicine | Hemophilia B - therapy | Hemophilia B - immunology | Genetic Vectors - administration & dosage | Transposases - metabolism | Humans | Mice, Inbred C57BL | Factor IX - genetics | Hepatocytes - pathology | Genetic Vectors - therapeutic use | Hepatocytes - metabolism | Organ Specificity | DNA Transposable Elements | Animals | Transposases - genetics | Mice | Disease Models, Animal | Genetic Therapy - methods | Index Medicus | Original
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7. Full Text Fetal gene therapy for neurodegenerative disease of infants
by Massaro, Giulia and Mattar, Citra N. Z and Wong, Andrew M. S and Sirka, Ernestas and Buckley, Suzanne M. K and Herbert, Bronwen R and Karlsson, Stefan and Perocheau, Dany P and Burke, Derek and Heales, Simon and Richard-Londt, Angela and Brandner, Sebastian and Huebecker, Mylene and Priestman, David A and Platt, Frances M and Mills, Kevin and Biswas, Arijit and Cooper, Jonathan D and Chan, Jerry K. Y and Cheng, Seng H and Waddington, Simon N and Rahim, Ahad A
Nature medicine, ISSN 1078-8956, 09/2018, Volume 24, Issue 9, pp. 1317 - 1323
Biochemistry & Molecular Biology | Life Sciences & Biomedicine | Medicine, Research & Experimental | Science & Technology | Cell Biology | Research & Experimental Medicine | Genetic Therapy | Animals | Gaucher Disease - genetics | Injections, Intravenous | Humans | Mice, Inbred C57BL | Fetus - metabolism | Injections, Intraventricular | Infant | Neurodegenerative Diseases - genetics | Neurodegenerative Diseases - therapy | Gaucher Disease - therapy | Usage | Care and treatment | Nervous system | Infants | Genetic aspects | Degeneration | Gene therapy | Health aspects | Neonates | Splenomegaly | Brain | Disease | Prophylaxis | Viruses | Recombinase | Dehydration | Neurodegeneration | Neck | Children | Ultrasound | Gaucher's disease | Enzymes | Strabismus | Fetuses | Lung diseases | Feasibility studies | Inflammation | Disease control | Patients | Cre recombinase | Neomycin | Hindbrain | Skin | Mice | Glucosylceramidase | Mutation | Index Medicus | Neurologi | Clinical Medicine | Neurology | Medical and Health Sciences | Klinisk medicin | Medicin och hälsovetenskap
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8. Full Text Production of lentiviral vectors using novel, enzymatically produced, linear DNA
by Karda, Rajvinder and Counsell, John R and Karbowniczek, Kinga and Caproni, Lisa J and Tite, John P and Waddington, Simon N
Gene therapy, ISSN 0969-7128, 2019, Volume 26, Issue 3-4, pp. 86 - 92
Biochemistry & Molecular Biology | Life Sciences & Biomedicine | Genetics & Heredity | Biotechnology & Applied Microbiology | Medicine, Research & Experimental | Science & Technology | Research & Experimental Medicine | Nucleic Acid Amplification Techniques - methods | Humans | Male | Genetic Vectors - genetics | DNA - genetics | Genetic Vectors - isolation & purification | Animals | Transfection | HEK293 Cells | Plasmids - genetics | Lentivirus - genetics | Mice | Genetic Therapy - methods | Technology application | Genetic vectors | Genes | Luciferase | DNA | DNA polymerases | Nucleotide sequencing | Gene therapy | DNA sequencing | Composition | Microbial genetic engineering | Equipment and supplies | Lentivirus | Methods | Neonates | Nucleotide sequence | Rodents | Deoxyribonucleic acid--DNA | Expression vectors | Gene expression
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