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One or more keywords matched the following items that are connected to Gray-Edwards, Heather
Item TypeName
Concept Genetic Therapy
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 Therapeutic response in feline sandhoff disease despite immunity to intracranial gene therapy.
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 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 Intravenous delivery of adeno-associated viral gene therapy in feline GM1 gangliosidosis.
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 AAV gene therapy for Tay-Sachs disease.
Academic Article Redosing Adeno-Associated Virus Gene Therapy to the Central Nervous System.
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 Adeno-associated virus-mediated gene therapy in a patient with Canavan disease using dual routes of administration and immune modulation.
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  • Gene therapy