| Item Type | Name |
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Concept
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Antigens, Ly
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|
Concept
|
Disease Models, Animal
|
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Concept
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Fibroblasts
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Concept
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Precision Medicine
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Concept
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Central Nervous System Diseases
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Concept
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Sandhoff Disease
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Concept
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Imaging, Three-Dimensional
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Concept
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Blood-Brain Barrier
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Concept
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beta-Hexosaminidase alpha Chain
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Concept
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Humans
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Concept
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Gangliosidoses, GM2
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Concept
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Brain Diseases
|
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Concept
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Genetic Therapy
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Concept
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Injections, Intraventricular
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Concept
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Hypothermia, Induced
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Concept
|
Magnetic Resonance Imaging
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Concept
|
Cross-Over Studies
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Concept
|
Gene Expression
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Concept
|
beta-Hexosaminidase beta Chain
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Concept
|
Genes, MHC Class II
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Concept
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Rare Diseases
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Concept
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Cats
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Concept
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Surgery, Computer-Assisted
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Concept
|
Disease Progression
|
|
Concept
|
Endothelium, Vascular
|
|
Concept
|
Injections
|
|
Concept
|
Dependovirus
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|
Concept
|
Treatment Outcome
|
|
Concept
|
Video Recording
|
|
Concept
|
Administration, Oral
|
|
Concept
|
Lysosomes
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|
Concept
|
Dogs
|
|
Concept
|
Brain Ischemia
|
|
Concept
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Algorithms
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|
Concept
|
Neurogenesis
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Concept
|
Cryotherapy
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|
Concept
|
Databases, Genetic
|
|
Concept
|
Stroke
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|
Concept
|
Tay-Sachs Disease
|
|
Concept
|
CHO Cells
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|
Concept
|
Injections, Spinal
|
|
Concept
|
Cricetulus
|
|
Concept
|
Genetic Predisposition to Disease
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|
Concept
|
Gangliosidosis, GM1
|
|
Concept
|
Area Under Curve
|
|
Concept
|
Mice
|
|
Concept
|
Thalamus
|
|
Concept
|
Growth Plate
|
|
Concept
|
Echocardiography
|
|
Concept
|
Carotid Artery, Internal
|
|
Concept
|
Electroencephalography
|
|
Concept
|
Cerebral Cortex
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|
Concept
|
Cattle
|
|
Concept
|
Transgenes
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|
Concept
|
Mice, Inbred C57BL
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|
Concept
|
Gene Expression Regulation
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|
Concept
|
Mutation
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|
Concept
|
Mice, Transgenic
|
|
Concept
|
Huntington Disease
|
|
Concept
|
Cerebellopontine Angle
|
|
Concept
|
Swine, Miniature
|
|
Concept
|
X-Ray Microtomography
|
|
Concept
|
Drug Administration Routes
|
|
Concept
|
Severity of Illness Index
|
|
Concept
|
Homeodomain Proteins
|
|
Concept
|
Primates
|
|
Concept
|
DNA, Viral
|
|
Concept
|
Collagen Type V
|
|
Concept
|
Adaptor Proteins, Signal Transducing
|
|
Concept
|
G(M1) Ganglioside
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|
Concept
|
Genes, Reporter
|
|
Concept
|
Survival Analysis
|
|
Concept
|
Green Fluorescent Proteins
|
|
Concept
|
G(M2) Ganglioside
|
|
Concept
|
Recombinant Fusion Proteins
|
|
Concept
|
Sheep
|
|
Concept
|
Delayed-Action Preparations
|
|
Concept
|
Maple Syrup Urine Disease
|
|
Concept
|
Leukocytes, Mononuclear
|
|
Concept
|
Transduction, Genetic
|
|
Concept
|
Genotype
|
|
Concept
|
Cerebrosides
|
|
Concept
|
Ehlers-Danlos Syndrome
|
|
Concept
|
Quantitative Trait Loci
|
|
Concept
|
Atrophy
|
|
Concept
|
Adenoviridae
|
|
Concept
|
Cisterna Magna
|
|
Concept
|
Case-Control Studies
|
|
Concept
|
beta-N-Acetylhexosaminidases
|
|
Concept
|
Mucopolysaccharidoses
|
|
Concept
|
Prospective Studies
|
|
Concept
|
Collagen
|
|
Concept
|
Sulfoglycosphingolipids
|
|
Concept
|
Tomography, X-Ray Computed
|
|
Concept
|
Cell Line
|
|
Concept
|
beta-Galactosidase
|
|
Concept
|
Polymerase Chain Reaction
|
|
Concept
|
Swine
|
|
Concept
|
Apoptosis Regulatory Proteins
|
|
Concept
|
United States Food and Drug Administration
|
|
Concept
|
Genetic Vectors
|
|
Concept
|
Gangliosides
|
|
Concept
|
Infarction, Middle Cerebral Artery
|
|
Concept
|
G(M2) Activator Protein
|
|
Concept
|
3-Methyl-2-Oxobutanoate Dehydrogenase (Lipoamide)
|
|
Concept
|
Immunohistochemistry
|
|
Concept
|
Gene Transfer Techniques
|
|
Academic Article
|
Widespread Central Nervous System Gene Transfer and Silencing After Systemic Delivery of Novel AAV-AS Vector.
|
|
Academic Article
|
In Vivo Selection Yields AAV-B1 Capsid for Central Nervous System and Muscle Gene Therapy.
|
|
Academic Article
|
Adeno-Associated Virus Gene Therapy in a Sheep Model of Tay-Sachs Disease.
|
|
Academic Article
|
Lipidomic Evaluation of Feline Neurologic Disease after AAV Gene Therapy.
|
|
Academic Article
|
AAV-mediated gene delivery attenuates neuroinflammation in feline Sandhoff disease.
|
|
Academic Article
|
Biomarkers for disease progression and AAV therapeutic efficacy in feline Sandhoff disease.
|
|
Academic Article
|
Novel Biomarkers of Human GM1 Gangliosidosis Reflect the Clinical Efficacy of Gene Therapy in a Feline Model.
|
|
Academic Article
|
Mucopolysaccharidosis-like phenotype in feline Sandhoff disease and partial correction after AAV gene therapy.
|
|
Academic Article
|
AAV-mediated gene delivery in a feline model of Sandhoff disease corrects lysosomal storage in the central nervous system.
|
|
Academic Article
|
Sustained normalization of neurological disease after intracranial gene therapy in a feline model.
|
|
Academic Article
|
High resolution MRI anatomy of the cat brain at 3 Tesla.
|
|
Academic Article
|
Therapeutic response in feline sandhoff disease despite immunity to intracranial gene therapy.
|
|
Academic Article
|
Widespread correction of central nervous system disease after intracranial gene therapy in a feline model of Sandhoff disease.
|
|
Academic Article
|
Focal cooling of brain parenchyma in a transient large vessel occlusion model: proof-of-concept.
|
|
Academic Article
|
A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system.
|
|
Academic Article
|
Pharmacokinetics of a commercially available product and a compounded formulation of extended-release levetiracetam after oral administration of a single dose in cats.
|
|
Academic Article
|
Ly6a Differential Expression in Blood-Brain Barrier Is Responsible for Strain Specific Central Nervous System Transduction Profile of AAV-PHP.B.
|
|
Academic Article
|
A Safe and Reliable Technique for CNS Delivery of AAV Vectors in the Cisterna Magna.
|
|
Academic Article
|
7T MRI Predicts Amelioration of Neurodegeneration in the Brain after AAV Gene Therapy.
|
|
Academic Article
|
Canine Models of Inherited Musculoskeletal and Neurodegenerative Diseases.
|
|
Academic Article
|
Volume and Infusion Rate Dynamics of Intraparenchymal Central Nervous System Infusion in a Large Animal Model.
|
|
Academic Article
|
Pronounced Therapeutic Benefit of a Single Bidirectional AAV Vector Administered Systemically in Sandhoff Mice.
|
|
Academic Article
|
Abnormal epiphyseal development in a feline model of Sandhoff disease.
|
|
Academic Article
|
Therapeutic benefit after intracranial gene therapy delivered during the symptomatic stage in a feline model of Sandhoff disease.
|
|
Academic Article
|
Large Animal Models of Huntington's Disease: What We Have Learned and Where We Need to Go Next.
|
|
Academic Article
|
Natural history study of glycan accumulation in large animal models of GM2 gangliosidoses.
|
|
Academic Article
|
PEA15 loss of function and defective cerebral development in the domestic cat.
|
|
Academic Article
|
A comprehensive study of a 29-capsid AAV library in a non-human primate central nervous system.
|
|
Academic Article
|
Intravenous delivery of adeno-associated viral gene therapy in feline GM1 gangliosidosis.
|
|
Academic Article
|
Natural history of Tay-Sachs disease in sheep.
|
|
Academic Article
|
Real-time MR tracking of AAV gene therapy with ?gal-responsive MR probe in a murine model of GM1-gangliosidosis.
|
|
Academic Article
|
Comparative route of administration studies using therapeutic siRNAs show widespread gene modulation in Dorset sheep.
|
|
Academic Article
|
Suppression of mutant C9orf72 expression by a potent mixed backbone antisense oligonucleotide.
|
|
Concept
|
Whole Genome Sequencing
|
|
Concept
|
Loss of Function Mutation
|
|
Concept
|
High-Throughput Nucleotide Sequencing
|
|
Concept
|
Neuroimaging
|
|
Concept
|
White Matter
|
|
Academic Article
|
Adeno-Associated Virus Delivery Limitations for Neurological Indications.
|
|
Academic Article
|
AAV gene therapy for Tay-Sachs disease.
|
|
Academic Article
|
Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders.
|
|
Academic Article
|
AAVrh10 vector corrects pathology in animal models of GM1 gangliosidosis and achieves widespread distribution in the CNS of nonhuman primates.
|
|
Academic Article
|
Redosing Adeno-Associated Virus Gene Therapy to the Central Nervous System.
|
|
Academic Article
|
Brain Alterations in Aged OVT73 Sheep Model of Huntington's Disease: An MRI Based Approach.
|
|
Academic Article
|
A pentasaccharide for monitoring pharmacodynamic response to gene therapy in GM1 gangliosidosis.
|
|
Academic Article
|
Life-Limiting Peripheral Organ Dysfunction in Feline Sandhoff Disease Emerges after Effective CNS Gene Therapy.
|
|
Academic Article
|
Precision medicine using whole genome sequencing in a cat identifies a novel COL5A1 variant for classical Ehlers-Danlos syndrome.
|
|
Academic Article
|
Levels of Synaptic Proteins in Brain and Neurofilament Light Chain in Cerebrospinal Fluid and Plasma of OVT73 Huntington's Disease Sheep Support a Prodromal Disease State.
|
|
Academic Article
|
Adeno-associated virus-mediated gene therapy in a patient with Canavan disease using dual routes of administration and immune modulation.
|
|
Academic Article
|
Awake intracerebroventricular delivery and safety assessment of oligonucleotides in a large animal model.
|
|
Academic Article
|
A Paradox of the Field's Own Success: Unintended Challenges in Bringing Cutting-Edge Science from the Bench to the Market.
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|
Academic Article
|
A minimally invasive endovascular approach to the cerebellopontine angle cistern enables broad CNS biodistribution of scAAV9-CB-GFP.
|
|
Academic Article
|
Neuroimaging Applications for the Delivery and Monitoring of Gene Therapy for Central Nervous System Diseases.
|
|
Academic Article
|
Quantitative reliability assessment of brain MRI volumetric measurements in type II GM1 gangliosidosis patients.
|
|
Academic Article
|
Quantifying and mitigating motor phenotypes induced by antisense oligonucleotides in the central nervous system.
|
|
Academic Article
|
Intravenous gene therapy improves lifespan and clinical outcomes in feline Sandhoff Disease.
|
|
Academic Article
|
Clinical and biochemical abnormalities in a feline model of GM2 activator deficiency.
|
|
Academic Article
|
Natural history progression of MRI brain volumetrics in type II late-infantile and juvenile GM1 gangliosidosis patients.
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|
Academic Article
|
BCKDHA-BCKDHB digenic gene therapy restores metabolic homeostasis in two mouse models and a calf with classic maple syrup urine disease.
|