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1.
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In vivo genome editing of the albumin locus as a platform for protein replacement therapy
Blood, ISSN 0006-4971, 10/2015, Volume 126, Issue 15, pp. 1777 - 1784
Life Sciences & Biomedicine | Hematology | Science & Technology | Hemophilia B - therapy | Dependovirus - genetics | Genetic Therapy | Transgenes - physiology | Genetic Vectors - administration & dosage | Fabry Disease - genetics | RNA Editing | Humans | Lysosomes - enzymology | Gaucher Disease - therapy | Promoter Regions, Genetic - genetics | Mucopolysaccharidosis I - therapy | Mucopolysaccharidosis II - therapy | Mucopolysaccharidosis II - genetics | Real-Time Polymerase Chain Reaction | Enzyme Replacement Therapy | Zinc Fingers | Albumins - metabolism | Liver - metabolism | Mice, Inbred C57BL | RNA, Messenger - genetics | Factor VIII - genetics | Factor IX - genetics | Hemophilia B - genetics | Hemophilia A - therapy | Reverse Transcriptase Polymerase Chain Reaction | Animals | Gaucher Disease - genetics | Endonucleases | Mucopolysaccharidosis I - genetics | Hemophilia A - genetics | High-Throughput Nucleotide Sequencing | Mice | Fabry Disease - therapy | Genome | Albumins - genetics | Index Medicus | Abridged Index Medicus | Gene Therapy
Journal Article
Movement disorders, ISSN 0885-3185, 05/2019, Volume 34, Issue 5, pp. 614 - 624
genetics | Parkinson's disease | movement disorders | lysosomal storage disease | Clinical Neurology | Neurosciences & Neurology | Life Sciences & Biomedicine | Science & Technology | Sphingomyelin Phosphodiesterase - genetics | Glucosylceramidase - genetics | Mucopolysaccharidosis III - genetics | Humans | Lysosomal Storage Diseases - genetics | Sandhoff Disease - genetics | Parkinson Disease - genetics | Phenotype | Gaucher Disease - genetics | Neuronal Ceroid-Lipofuscinoses - genetics | Proton-Translocating ATPases - genetics | Galactosylceramidase - genetics | Adult | Parkinsonian Disorders - genetics | Leukodystrophy, Globoid Cell - genetics | Mutation | Child | Niemann-Pick Diseases - genetics | Pediatrics | Genetics | Children | Epidemiology | Risk factors | Cells | Diseases | Basal ganglia | Neurodegenerative diseases | Central nervous system diseases | Homeostasis | Lysosomes | Lysosomal storage diseases | Neurological diseases | Evolution | Glucosylceramidase | Movement disorders | Gaucher's disease | Index Medicus | Parkinson’s Disease | Lysosomal Storage Disease | Movement Disorders
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The molecular basis of autosomal recessive diseases among the Arabs and Druze in Israel
Human genetics, ISSN 0340-6717, 11/2010, Volume 128, Issue 5, pp. 473 - 479
Human Genetics | Gene Function | Molecular Medicine | Biomedicine | Metabolic Diseases | Life Sciences & Biomedicine | Genetics & Heredity | Science & Technology | Fundamental and applied biological sciences. Psychology | Classical genetics, quantitative genetics, hybrids | Human | Biological and medical sciences | Genetics of eukaryotes. Biological and molecular evolution | Maple Syrup Urine Disease - genetics | Humans | Genetic Diseases, Inborn - genetics | Hypoparathyroidism - genetics | Christianity | Genetic Diseases, Inborn - epidemiology | Genes, Recessive | Intellectual Disability - genetics | Epidermolysis Bullosa - genetics | Leukodystrophy, Metachromatic - genetics | Islam | Israel - epidemiology | Xanthomatosis, Cerebrotendinous - genetics | Albinism - genetics | Arabs - genetics | Ataxia Telangiectasia - genetics | Bardet-Biedl Syndrome - genetics | Mucopolysaccharidosis I - genetics | Leukodystrophy, Globoid Cell - genetics | Mutation | Druses | Deafness | Genetic disorders | Index Medicus
Journal Article
Molecular therapy, ISSN 1525-0016, 03/2006, Volume 13, Issue 3, pp. 528 - 537
Vector transport | β-glucuronidase | MPS VII | Serotype | Capsid | Lysosomal storage disease | Genetics & Heredity | Life Sciences & Biomedicine | Biotechnology & Applied Microbiology | Medicine, Research & Experimental | Science & Technology | Research & Experimental Medicine | Dependovirus - genetics | Genetic Therapy | Axons - enzymology | Capsid - classification | Humans | Capsid - enzymology | RNA, Messenger - metabolism | Brain - metabolism | Mucopolysaccharidosis VII - virology | Virus Assembly - genetics | Mucopolysaccharidosis VII - genetics | Serotyping | Disease Models, Animal | Transduction, Genetic | Genetic Vectors - immunology | Capsid Proteins - metabolism | Mucopolysaccharidosis VII - therapy | Genetic Vectors - genetics | Mice, Inbred C3H | Animals | Glucuronidase - biosynthesis | Glucuronidase - genetics | Genetic Vectors - biosynthesis | Mice | Axons - virology | Capsid Proteins - biosynthesis | Capsid Proteins - genetics | Virus diseases | Enzymes | Brain damage | Viral proteins | Neurons | Viral genetics | Index Medicus
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Human molecular genetics, ISSN 0964-6906, 1/2008, Volume 17, Issue 1, pp. 119 - 129
Biochemistry & Molecular Biology | Genetics & Heredity | Life Sciences & Biomedicine | Science & Technology | Fundamental and applied biological sciences. Psychology | Biological and medical sciences | Molecular and cellular biology | Genetics of eukaryotes. Biological and molecular evolution | Lysosomal Storage Diseases, Nervous System - physiopathology | Microtubule-Associated Proteins - genetics | Lysosomal Storage Diseases - physiopathology | Microtubule-Associated Proteins - metabolism | Humans | Mucopolysaccharidosis III - pathology | Nerve Degeneration - physiopathology | Nerve Degeneration - genetics | Autophagy - physiology | DNA Primers - genetics | Lysosomal Storage Diseases, Nervous System - pathology | Ubiquitination | Transfection | Base Sequence | Multiple Sulfatase Deficiency Disease - pathology | Lysosomes - pathology | Autophagy - genetics | Mucopolysaccharidosis III - physiopathology | Membrane Fusion | Mucopolysaccharidosis III - genetics | Cells, Cultured | Lysosomal Storage Diseases - genetics | Mitochondria - pathology | Lysosomal Storage Diseases, Nervous System - genetics | Phagosomes - pathology | Lysosomal Storage Diseases - pathology | Nerve Degeneration - pathology | Multiple Sulfatase Deficiency Disease - genetics | Animals | Mice | Multiple Sulfatase Deficiency Disease - physiopathology | Index Medicus
Journal Article
Journal of Medical Genetics, ISSN 0022-2593, 07/2012, Volume 49, Issue 7, pp. 455 - 461
Life Sciences & Biomedicine | Genetics & Heredity | Science & Technology | Fundamental and applied biological sciences. Psychology | Biological and medical sciences | Molecular and cellular biology | Genetics of eukaryotes. Biological and molecular evolution | Medical sciences | Medical genetics | Humans | Child, Preschool | Cytoplasm - metabolism | Male | rab GTP-Binding Proteins - genetics | Mucopolysaccharidosis IV - genetics | Genetic Loci | Intellectual Disability - genetics | Exome | Osteochondrodysplasias - genetics | Adult | Female | Genetic Diseases, X-Linked - genetics | Cytoplasm - genetics | Child | Genetic Linkage | rab GTP-Binding Proteins - metabolism | Fluorescent Antibody Technique - methods | Genotype | Genes, Recessive | Genetic Heterogeneity | Dwarfism - genetics | Mucopolysaccharidosis IV - physiopathology | Osteochondrodysplasias - congenital | Pedigree | Golgi Apparatus - metabolism | Consanguinity | Mutation | Golgi Apparatus - genetics | Index Medicus
Journal Article
Human gene therapy, ISSN 1043-0342, 03/2014, Volume 25, Issue 3, pp. 199 - 211
Research Articles | Genetics & Heredity | Life Sciences & Biomedicine | Biotechnology & Applied Microbiology | Medicine, Research & Experimental | Science & Technology | Research & Experimental Medicine | Genetic Therapy | Genetic Vectors - administration & dosage | Glucuronidase - metabolism | Lysosomes - enzymology | Behavior, Animal | Brain - metabolism | Microglia - immunology | Helper Viruses | Adenoviruses, Canine - genetics | Mucopolysaccharidosis VII - genetics | Transgenes | Disease Models, Animal | Gene Expression | Genetic Vectors - immunology | Glycosaminoglycans - metabolism | Mucopolysaccharidosis VII - therapy | Immunity, Innate | Genetic Vectors - genetics | Injections | Animals | Glucuronidase - genetics | Dogs | Brain - pathology | Mice | Enzyme Activation | Brain - immunology | Index Medicus | Life Sciences | Biochemistry, Molecular Biology
Journal Article
Human gene therapy, ISSN 1043-0342, 09/2019, Volume 30, Issue 9, pp. 152 - 1066
Research Articles | Genetics & Heredity | Life Sciences & Biomedicine | Biotechnology & Applied Microbiology | Medicine, Research & Experimental | Science & Technology | Research & Experimental Medicine | Dependovirus - genetics | Hydrolases - genetics | Genetic Vectors - administration & dosage | Corpus Striatum - metabolism | Organ Specificity - genetics | Mucopolysaccharidosis III - metabolism | Neurons - metabolism | Transgenes | Gene Order | Disease Models, Animal | Gene Expression | Mucopolysaccharidosis III - physiopathology | Transduction, Genetic | Cytokines - metabolism | Mucopolysaccharidosis III - genetics | Treatment Outcome | Genetic Vectors - genetics | Genetic Vectors - isolation & purification | Animals | Fluorescent Antibody Technique | Mucopolysaccharidosis III - therapy | Biomarkers | Mice | Enzyme Activation | Genetic Therapy - adverse effects | Genetic Therapy - methods | Exchanging | Heparan sulfate | Enzymes | Level (quantity) | Enzyme activity | Poultry | Clinical trials | Trinucleotide repeats | Fluorescence | Lysosomal storage diseases | Viruses | Cognition | Mucopolysaccharidosis | Injection | Phosphoglycerate kinase | Sulfates | Kinases | Gene expression | Enzymatic activity | Neurodegeneration | Actin | Green fluorescent protein | Gene therapy | Expression vectors | Index Medicus
Journal Article
Human gene therapy, ISSN 1043-0342, 06/2017, Volume 28, Issue 6, pp. 45 - 463
Review Article | gene therapy | AAV vectors | ITR, stem cell | stem cell therapy | Genetics & Heredity | Life Sciences & Biomedicine | Biotechnology & Applied Microbiology | Medicine, Research & Experimental | Science & Technology | Research & Experimental Medicine | Genetic Therapy - ethics | Parkinson Disease - therapy | Cell- and Tissue-Based Therapy - methods | Dependovirus - genetics | Immunologic Deficiency Syndromes - pathology | Stem Cells - immunology | Humans | Transplantation, Autologous | Stem Cells - cytology | Stem Cells - metabolism | Anemia, Aplastic - therapy | Mucopolysaccharidosis I - therapy | Immunologic Deficiency Syndromes - immunology | Transduction, Genetic - methods | Anemia, Aplastic - genetics | Anemia, Aplastic - pathology | Immunologic Deficiency Syndromes - therapy | Parkinson Disease - pathology | Dependovirus - metabolism | Genetic Vectors - chemistry | Mucopolysaccharidosis I - pathology | Parkinson Disease - immunology | Genetic Vectors - metabolism | Multiple Sclerosis - genetics | Parkinson Disease - genetics | Multiple Sclerosis - therapy | Mucopolysaccharidosis I - immunology | Stem Cell Transplantation - methods | Immunologic Deficiency Syndromes - genetics | Multiple Sclerosis - immunology | Multiple Sclerosis - pathology | Mucopolysaccharidosis I - genetics | Transduction, Genetic - standards | Anemia, Aplastic - immunology | Genetic Therapy - methods | Cell culture | Graft-versus-host reaction | Therapy | Multiple sclerosis | Parkinson's disease | Disease | Toxicity | Stem cell transplantation | Viruses | Lymphocytes T | Mucopolysaccharidosis | Infusion | Neurotoxicity | Immunology | Lymphocytes | Movement disorders | Expression vectors | Autografts | Neurodegenerative diseases | Gene transfer | Anemia | Therapeutic applications | Medical treatment | Immunodeficiency | Aplastic anemia | Nucleic acids | Patients | Stem cells | Index Medicus | Review | stem cell | ITR
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