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Adeno-associated virus-delivered artificial microRNA extends survival and delays paralysis in an amyotrophic lateral sclerosis mouse model

Annals of neurology, 2016-04, Vol.79 (4), p.687-700

2016

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Spinal projections of the A5, A6 (locus coeruleus), and A7 noradrenergic cell groups in rats

Journal of comparative neurology (1911), 2012-06, Vol.520 (9), p.1985-2001

2012

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Life‐Limiting Peripheral Organ Dysfunction in Feline Sandhoff Disease Emerges after Effective CNS Gene Therapy

Annals of neurology, 2023-11, Vol.94 (5), p.969-986

2023

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Cerebral organoids derived from Sandhoff disease-induced pluripotent stem cells exhibit impaired neurodifferentiation[S]

Journal of lipid research, 2018-03, Vol.59 (3), p.550-563

2018

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Bis(monoacylglycero)phosphate: a secondary storage lipid in the gangliosidoses

Journal of lipid research, 2015-05, Vol.56 (5), p.1005-1006

2015

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Selection of an Efficient AAV Vector for Robust CNS Transgene Expression

Molecular therapy. Methods & clinical development, 2019-12, Vol.15, p.320-332

2019

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Gene Transfer to the CNS Using Recombinant Adeno‐Associated Virus

Current protocols in microbiology (Online), 2013-05, Vol.29 (1), p.14D.5.1-14D.5.18

2013

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Expression of ALS-PFN1 impairs vesicular degradation in iPSC-derived microglia

Nature communications, 2024-03, Vol.15 (1), p.2497-2497, Article 2497

2024

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Mutant Huntingtin Impairs Vesicle Formation from Recycling Endosomes by Interfering with Rab11 Activity

Molecular and Cellular Biology, 2009-11, Vol.29 (22), p.6106-6116

2009

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EZH2 inhibition reactivates epigenetically silenced FMR1 and normalizes molecular and electrophysiological abnormalities in fragile X syndrome neurons

Frontiers in neuroscience, 2024-02, Vol.18, p.1348478-1348478

2024

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[18F]Fluoropyridine‐losartan: A new approach toward human Positron Emission Tomography imaging of Angiotensin II Type 1 receptors

Journal of labelled compounds & radiopharmaceuticals, 2023-03, Vol.66 (3), p.73-85

2023

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Sustained normalization of neurological disease after intracranial gene therapy in a feline model

Science translational medicine, 2014-04, Vol.6 (231), p.231ra48

2014

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Adeno-Associated Virus Neutralizing Antibodies in Large Animals and Their Impact on Brain Intraparenchymal Gene Transfer

Molecular therapy. Methods & clinical development, 2018-12, Vol.11, p.65-72

2018

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Upregulating β-hexosaminidase activity in rodents prevents α-synuclein lipid associations and protects dopaminergic neurons from α-synuclein-mediated neurotoxicity

Acta neuropathologica communications, 2020-08, Vol.8 (1), p.127-14, Article 127

2020

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AAVrh10 vector corrects pathology in animal models of GM1 gangliosidosis and achieves widespread distribution in the CNS of nonhuman primates

Molecular therapy. Methods & clinical development, 2022-12, Vol.27, p.281-292

2022

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Gaussia Luciferase Variant for High-Throughput Functional Screening Applications

Analytical chemistry (Washington), 2009-08, Vol.81 (16), p.7102-7106

2009

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Awake intracerebroventricular delivery and safety assessment of oligonucleotides in a large animal model

Molecular therapy. Methods & clinical development, 2023-12, Vol.31, p.101122-101122, Article 101122

2023

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Long-Term Therapeutic Efficacy of Intravenous AAV-Mediated Hamartin Replacement in Mouse Model of Tuberous Sclerosis Type 1

Molecular therapy. Methods & clinical development, 2019-12, Vol.15, p.18-26

2019

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Production of High-Titer Retrovirus and Lentivirus Vectors

Cold Spring Harbor protocols, 2018-04, Vol.2018 (4), p.pdb.prot095687

2018

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Titration of Lentivirus Vectors

Cold Spring Harbor protocols, 2018-04, Vol.2018 (4), p.pdb.prot095695

2018

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Real-time MR tracking of AAV gene therapy with βgal-responsive MR probe in a murine model of GM1-gangliosidosis

Molecular therapy. Methods & clinical development, 2021-12, Vol.23, p.128-134

2021

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Production of Recombinant Adeno-Associated Viruses (rAAVs) by Transient Transfection

Cold Spring Harbor protocols, 2020-02, Vol.2020 (2), p.pdb.prot095596

2020

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7T MRI Predicts Amelioration of Neurodegeneration in the Brain after AAV Gene Therapy

Molecular therapy. Methods & clinical development, 2020-06, Vol.17, p.258-270

2020

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A vida de Santo Ildefonso e o imaginário ligado ao culto Mariano no Reino Visigodo de Toledo (séculos VII-VIII)nto Ildefonso e o imaginário ligado ao culto mariano no Reino Visigodo de Toledo ( séculos VII- VIII)

Acta Scientiarum : education, 2019-10, Vol.41 (1), p.e48156

Esteves, Germano Miguel Favaro

2019

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Adeno-associated virus-delivered artificial microRNA extends survival and delays paralysis in an amyotrophic lateral sclerosis mouse model

Spinal projections of the A5, A6 (locus coeruleus), and A7 noradrenergic cell groups in rats

Life‐Limiting Peripheral Organ Dysfunction in Feline Sandhoff Disease Emerges after Effective CNS Gene Therapy

A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system

Cerebral organoids derived from Sandhoff disease-induced pluripotent stem cells exhibit impaired neurodifferentiation[S]

Bis(monoacylglycero)phosphate: a secondary storage lipid in the gangliosidoses

Selection of an Efficient AAV Vector for Robust CNS Transgene Expression

Gene Transfer to the CNS Using Recombinant Adeno‐Associated Virus

Expression of ALS-PFN1 impairs vesicular degradation in iPSC-derived microglia

Mutant Huntingtin Impairs Vesicle Formation from Recycling Endosomes by Interfering with Rab11 Activity

EZH2 inhibition reactivates epigenetically silenced FMR1 and normalizes molecular and electrophysiological abnormalities in fragile X syndrome neurons

[18F]Fluoropyridine‐losartan: A new approach toward human Positron Emission Tomography imaging of Angiotensin II Type 1 receptors

Sustained normalization of neurological disease after intracranial gene therapy in a feline model

Adeno-Associated Virus Neutralizing Antibodies in Large Animals and Their Impact on Brain Intraparenchymal Gene Transfer

Upregulating β-hexosaminidase activity in rodents prevents α-synuclein lipid associations and protects dopaminergic neurons from α-synuclein-mediated neurotoxicity

AAVrh10 vector corrects pathology in animal models of GM1 gangliosidosis and achieves widespread distribution in the CNS of nonhuman primates

Gaussia Luciferase Variant for High-Throughput Functional Screening Applications

Awake intracerebroventricular delivery and safety assessment of oligonucleotides in a large animal model

Long-Term Therapeutic Efficacy of Intravenous AAV-Mediated Hamartin Replacement in Mouse Model of Tuberous Sclerosis Type 1

Production of High-Titer Retrovirus and Lentivirus Vectors

Titration of Lentivirus Vectors

Real-time MR tracking of AAV gene therapy with βgal-responsive MR probe in a murine model of GM1-gangliosidosis

Production of Recombinant Adeno-Associated Viruses (rAAVs) by Transient Transfection

7T MRI Predicts Amelioration of Neurodegeneration in the Brain after AAV Gene Therapy

A vida de Santo Ildefonso e o imaginário ligado ao culto Mariano no Reino Visigodo de Toledo (séculos VII-VIII)nto Ildefonso e o imaginário ligado ao culto mariano no Reino Visigodo de Toledo ( séculos VII- VIII)

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