Miguel Sena-Esteves Selected Research

Sandhoff Disease (Sandhoff's Disease)

1/2021	Therapeutic benefit after intracranial gene therapy delivered during the symptomatic stage in a feline model of Sandhoff disease.
12/2020	Abnormal epiphyseal development in a feline model of Sandhoff disease.
1/2020	Upregulating β-hexosaminidase activity in rodents prevents α-synuclein lipid associations and protects dopaminergic neurons from α-synuclein-mediated neurotoxicity.
1/2018	Cerebral organoids derived from Sandhoff disease-induced pluripotent stem cells exhibit impaired neurodifferentiation.
1/2017	AAV-mediated gene delivery attenuates neuroinflammation in feline Sandhoff disease.
9/2015	Mucopolysaccharidosis-like phenotype in feline Sandhoff disease and partial correction after AAV gene therapy.
5/2015	Bis(monoacylglycero)phosphate: a secondary storage lipid in the gangliosidoses.
3/2015	AAV-mediated gene delivery in a feline model of Sandhoff disease corrects lysosomal storage in the central nervous system.
1/2015	Biomarkers for disease progression and AAV therapeutic efficacy in feline Sandhoff disease.

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Miguel Sena-Esteves Research Topics

Disease

10	Neoplasms (Cancer) 12/2019 - 10/2008
9	Sandhoff Disease (Sandhoff's Disease) 01/2021 - 01/2015
8	Neurodegenerative Diseases (Neurodegenerative Disease) 01/2022 - 01/2015
6	GM1 Gangliosidosis (Gangliosidosis GM1) 01/2022 - 04/2014
6	Lysosomal Storage Diseases (Lysosomal Storage Disease) 01/2021 - 10/2010
5	Disease Progression 01/2022 - 01/2015
5	Tay-Sachs Disease 01/2022 - 01/2015
5	Glioma (Gliomas) 01/2018 - 12/2004
5	Brain Neoplasms (Brain Tumor) 01/2018 - 10/2008
4	Body Weight (Weight, Body) 01/2022 - 02/2010
4	Nervous System Diseases (Neurological Disorders) 01/2022 - 08/2011
4	Huntington Disease (Huntington's Disease) 01/2019 - 12/2010
3	Intellectual Disability (Idiocy) 12/2022 - 01/2013
3	Tuberous Sclerosis (Bourneville's Disease) 12/2019 - 01/2013
3	GM2 Gangliosidoses (GM2 Gangliosidosis) 01/2018 - 01/2014
3	Obesity 01/2014 - 12/2007
2	Hydrocephalus (Hydrocephaly) 12/2019 - 01/2013
2	Epilepsy (Aura) 12/2019 - 01/2013
2	Neurologic Manifestations (Neurological Manifestations) 12/2019 - 01/2013
2	Autistic Disorder (Autism) 12/2019 - 01/2013
2	Astrocytoma (Pilocytic Astrocytoma) 12/2019 - 01/2013
2	Infections 12/2018 - 05/2009
2	Alzheimer Disease (Alzheimer's Disease) 12/2017 - 06/2007
2	Brain Diseases (Brain Disorder) 07/2017 - 01/2013
2	Amyotrophic Lateral Sclerosis (Lou Gehrig's Disease) 01/2017 - 04/2016
2	Glioblastoma (Glioblastoma Multiforme) 01/2017 - 12/2008
2	Gangliosidoses (Gangliosidosis) 05/2015 - 01/2014
2	Rabies (Hydrophobia) 12/2004 - 12/2004
1	Seizures (Absence Seizure) 01/2022
1	Polyps 01/2022
1	Glycogen Storage Disease Type II (Pompe's Disease) 01/2019
1	Metabolic Diseases (Metabolic Disease) 01/2018
1	Memory Disorders (Memory Loss) 12/2017
1	Lung Diseases (Lung Disease) 01/2017
1	Paralysis (Palsy) 04/2016
1	Neuroinflammatory Diseases 04/2016
1	Weight Gain 10/2015
1	Hyperphagia (Overeating) 01/2014
1	Fatty Liver 01/2014
1	Amyloidosis 08/2013
1	Hepatocellular Carcinoma (Hepatoma) 08/2013
1	Neurilemmoma (Schwannoma) 02/2013
1	Hamartoma 01/2013
1	Frontotemporal Dementia (Semantic Dementia) 10/2012

Drug/Important Bio-Agent (IBA)

11	EnzymesIBA 12/2022 - 06/2007
10	Proteins (Proteins, Gene)FDA Link 12/2019 - 06/2007
7	G(M2) Ganglioside (Ganglioside GM2)IBA 01/2021 - 03/2015
6	GalactosidasesIBA 01/2022 - 10/2010
5	Messenger RNA (mRNA)IBA 01/2019 - 12/2008
4	Biomarkers (Surrogate Marker)IBA 01/2022 - 12/2008
3	Hexosaminidase A (Hex A)IBA 01/2022 - 01/2018
3	trans-sodium crocetinate (crocetin)IBA 12/2019 - 01/2013
3	AcidsIBA 01/2019 - 10/2010
3	Interferon-betaIBA 09/2018 - 10/2008
3	MicroRNAs (MicroRNA)IBA 01/2018 - 12/2008
3	Brain-Derived Neurotrophic Factor (BDNF)IBA 01/2014 - 12/2007
2	Conditioned Culture MediaIBA 01/2022 - 08/2013
2	Hexosaminidases (Hexosaminidase)IBA 01/2020 - 05/2015
2	Tuberous Sclerosis Complex 1 ProteinIBA 12/2019 - 10/2015
2	GangliosidesIBA 05/2015 - 01/2014
2	LuciferasesIBA 02/2013 - 08/2009
2	Glycoproteins (Glycoprotein)IBA 12/2004 - 09/2002
1	Protein Disulfide-IsomerasesIBA 12/2022
1	DisulfidesIBA 12/2022
1	Anticonvulsants (Antiepileptic Drugs)IBA 01/2022
1	Biopolymers (Biopolymer)IBA 01/2022
1	PolyphosphatesIBA 01/2022
1	Therapeutic UsesIBA 01/2020
1	Tuberous Sclerosis Complex 2 ProteinIBA 12/2019
1	G(M1) GangliosideIBA 12/2019
1	alpha-Glucosidases (Acid Maltase)IBA 01/2019
1	Small Interfering RNA (siRNA)IBA 01/2019
1	GlycogenIBA 01/2019
1	CytokinesIBA 09/2018
1	Lysergic Acid Diethylamide (LSD)IBA 01/2018
1	Transcription Factors (Transcription Factor)IBA 12/2017
1	GlycolipidsIBA 09/2017
1	SphingolipidsIBA 09/2017
1	RNA (Ribonucleic Acid)IBA 07/2017
1	Superoxide Dismutase-1IBA 01/2017
1	Firefly LuciferasesIBA 11/2016
1	LipidsIBA 05/2015
1	bis(monoacylglyceryl)phosphateIBA 05/2015
1	Fatty Acids (Saturated Fatty Acids)IBA 05/2015
1	Apoptosis Regulatory ProteinsIBA 10/2014
1	CD36 AntigensIBA 01/2014
1	Glucose (Dextrose)FDA LinkGeneric 01/2014
1	trkB ReceptorIBA 01/2014
1	Gabapentin (Neurontin)FDA LinkGeneric 01/2014
1	Calcium Channels (Calcium Channel)IBA 01/2014
1	Tumor Necrosis Factor-alpha (Tumor Necrosis Factor)IBA 06/2013
1	LigandsIBA 06/2013
1	Death Domain ReceptorsIBA 06/2013
1	IceIBA 02/2013
1	Cre recombinaseIBA 01/2013
1	ProgranulinsIBA 10/2012

Therapy/Procedure

11	Therapeutics 01/2022 - 10/2008
3	Injections 01/2020 - 11/2010
1	Aftercare (After-Treatment) 01/2022
1	Transplantation 01/2018