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Sena-Esteves, Miguel
One or more keywords matched the following properties of Sena-Esteves, Miguel
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The Sena-Esteves laboratory is investigating gene therapy approaches for the treatment of neurodegenerative lysosomal storage diseases such as GM1-gangliosidosis and GM2-gangliosidoses (Tay-Sachs and Sandhoff diseases). We have devised new ways to deliver therapeutic levels of the missing enzymes to the entire brain by injection of adeno-associated virus (AAV) vectors into specific structures in the CNS. Based on the exceptional results that we have obtained in animal models, we are initiating pre-clinical studies that will culminate in a human clinical trial for Tay-Sachs disease. Similar work is planned for GM1-gangliosidosis.

Also we are working on developing new AAV vectors capable of crossing the blood brain barrier for effective gene delivery to the brain after intravascular infusion. We have introduced a new paradigm for brain tumor gene therapy based on the genetic modification of normal brain to create an environment, which is non-permissive to tumor growth. We have demonstrated the effectiveness of this approach for preventing the formation and precluding growth of brain tumors using xenograft models in nude mice. Also we are initiating studies directed at understanding the contribution of different genes to the migratory behavior of GBM cells and strategies to counteract it. Finally we are investigating new gene therapy approaches for Ataxia Telangiectasia.
Summary Dr. Esteves is a neuroscientist with interest in gene therapy to help treat neurodegenerative diseases.
One or more keywords matched the following items that are connected to Sena-Esteves, Miguel
Item TypeName
Academic Article Switching on the lights for gene therapy.
Academic Article Several rAAV vectors efficiently cross the blood-brain barrier and transduce neurons and astrocytes in the neonatal mouse central nervous system.
Academic Article Gene transfer to the CNS using recombinant adeno-associated virus.
Concept Genetic Therapy
Academic Article Systemic AAV9 gene transfer in adult GM1 gangliosidosis mice reduces lysosomal storage in CNS and extends lifespan.
Academic Article Adeno-associated virus-delivered artificial microRNA extends survival and delays paralysis in an amyotrophic lateral sclerosis mouse model.
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 Systemic AAV9-IFN? gene delivery treats highly invasive glioblastoma.
Academic Article Intracranial AAV-IFN-? gene therapy eliminates invasive xenograft glioblastoma and improves survival in orthotopic syngeneic murine model.
Academic Article Direct Intracranial Injection of AAVrh8 Encoding Monkey ?-N-Acetylhexosaminidase Causes Neurotoxicity in the Primate Brain.
Academic Article Systemic Delivery of AAVB1-GAA Clears Glycogen and Prolongs Survival in a Mouse Model of Pompe Disease.
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 Selective Neuronal Uptake and Distribution of AAVrh8, AAV9, and AAVrh10 in Sheep After Intra-Striatal Administration.
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 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 AAV9-Mediated Intrastriatal Delivery of GNAO1 Reduces Hyperlocomotion in Gnao1 Heterozygous R209H Mutant Mice.
Academic Article Genetic therapy for the nervous system.
Academic Article Gene therapy: charting a future course--summary of a National Institutes of Health Workshop, April 12, 2013.
Academic Article Gene therapy for the nervous system: challenges and new strategies.
Academic Article Microvesicle-associated AAV vector as a novel gene delivery system.
Academic Article Healing genes in the nervous system.
Academic Article Human GLB1 knockout cerebral organoids: A model system for testing AAV9-mediated GLB1 gene therapy for reducing GM1 ganglioside storage in GM1 gangliosidosis.
Academic Article Regression of schwannomas induced by adeno-associated virus-mediated delivery of caspase-1.
Academic Article Critical issues in gene therapy for neurologic disease.
Academic Article Mutant sodium channel for tumor therapy.
Academic Article Survival benefit and phenotypic improvement by hamartin gene therapy in a tuberous sclerosis mouse brain model.
Academic Article Transduction of satellite cells after prenatal intramuscular administration of lentiviral vectors.
Academic Article Generation of stable retrovirus packaging cell lines after transduction with herpes simplex virus hybrid amplicon vectors.
Academic Article Evaluation and optimization of the administration of recombinant adeno-associated viral vectors (serotypes 2/1, 2/2, 2/rh8, 2/9, and 2/rh10) by convection-enhanced delivery to the striatum.
Academic Article Gene therapy for the central nervous system in the lysosomal storage disorders.
Academic Article Adeno Associated Viral Vector Delivered RNAi for Gene Therapy of SOD1 Amyotrophic Lateral Sclerosis.
Academic Article Virus vector-mediated genetic modification of brain tumor stromal cells after intravenous delivery.
Academic Article Viral vectors for therapy of neurologic diseases.
Academic Article Intravenous gene therapy improves lifespan and clinical outcomes in feline Sandhoff Disease.
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  • Gene therapy