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Complete Correction of Brain and Spinal Cord Pathology in Metachromatic Leukodystrophy Mice.
- Published in:
- Frontiers in Molecular Neuroscience, 2021, v. 13, p. 1, doi. 10.3389/fnmol.2021.677895
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- Publication type:
- Article
Gene therapy for leukodystrophies.
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- Human Molecular Genetics, 2011, v. 20, n. R1, p. R42, doi. 10.1093/hmg/ddr142
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- Publication type:
- Article
Valproic acid induces antioxidant effects in X-linked adrenoleukodystrophy.
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- Human Molecular Genetics, 2010, v. 19, n. 10, p. 2005, doi. 10.1093/hmg/ddq082
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- Publication type:
- Article
Efficient intracerebral delivery of AAV5 vector encoding human ARSA in non-human primate.
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- Human Molecular Genetics, 2010, v. 19, n. 1, p. 147, doi. 10.1093/hmg/ddp475
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- Publication type:
- Article
Intracerebral adeno-associated virus-mediated gene transfer in rapidly progressive forms of metachromatic leukodystrophy.
- Published in:
- Human Molecular Genetics, 2006, v. 15, n. 1, p. 53, doi. 10.1093/hmg/ddi425
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- Publication type:
- Article
CYP46A1 gene therapy deciphers the role of brain cholesterol metabolism in Huntington's disease.
- Published in:
- 2019
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- Publication type:
- journal article
Reply: Beneficial effect of interleukin-2-based immunomodulation in Alzheimer-like pathology.
- Published in:
- 2017
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- Publication type:
- letter
Interleukin-2 improves amyloid pathology, synaptic failure and memory in Alzheimer's disease mice.
- Published in:
- 2017
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- Publication type:
- journal article
Restoring brain cholesterol turnover improves autophagy and has therapeutic potential in mouse models of spinocerebellar ataxia.
- Published in:
- Acta Neuropathologica, 2019, v. 138, n. 5, p. 837, doi. 10.1007/s00401-019-02019-7
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- Publication type:
- Article
Viral gene transfer of APPsα rescues synaptic failure in an Alzheimer's disease mouse model.
- Published in:
- Acta Neuropathologica, 2016, v. 131, n. 2, p. 247, doi. 10.1007/s00401-015-1498-9
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- Publication type:
- Article
The role of microglia in human disease: therapeutic tool or target?
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- Acta Neuropathologica, 2014, v. 128, n. 3, p. 363, doi. 10.1007/s00401-014-1330-y
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- Publication type:
- Article
Genetically modified macrophages accelerate myelin repair.
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- EMBO Molecular Medicine, 2022, v. 14, n. 8, p. 1, doi. 10.15252/emmm.202114759
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- Publication type:
- Article
Lentiviral vector-mediated overexpression of mutant ataxin-7 recapitulates SCA7 pathology and promotes accumulation of the FUS/TLS and MBNL1 RNA-binding proteins.
- Published in:
- Molecular Neurodegeneration, 2016, v. 11, p. 1, doi. 10.1186/s13024-016-0123-2
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- Publication type:
- Article
Alzheimer's disease-like APP processing in wild-type mice identifies synaptic defects as initial steps of disease progression.
- Published in:
- Molecular Neurodegeneration, 2016, v. 11, p. 1, doi. 10.1186/s13024-016-0070-y
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- Publication type:
- Article
Increasing membrane cholesterol of neurons in culture recapitulates Alzheimer's disease early phenotypes.
- Published in:
- Molecular Neurodegeneration, 2014, v. 9, n. 1, p. 1, doi. 10.1186/1750-1326-9-60
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- Publication type:
- Article
Adeno-associated Virus Gene Therapy With Cholesterol 24-Hydroxylase Reduces the Amyloid Pathology Before or After the Onset of Amyloid Plaques in Mouse Models of Alzheimer's Disease.
- Published in:
- Molecular Therapy, 2010, v. 18, n. 1, p. 44, doi. 10.1038/mt.2009.175
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- Publication type:
- Article
429. AAV5-Mediated Delivery of Human Aryl Sulfatase A (hARSA) Prevents Sufatide Storage and Neuropathological Phenotype in Metachromatic Leukodystrophy (MLD) Mice
- Published in:
- 2005
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- Publication type:
- Abstract
Transduced CD34<sup>+</sup> cells from adrenoleukodystrophy patients with HIV-derived vector mediate long-term engraftment of NOD/SCID mice
- Published in:
- Molecular Therapy, 2003, v. 7, n. 3, p. 317, doi. 10.1016/S1525-0016(03)00002-9
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- Publication type:
- Article
The APP Intracellular Domain Is Required for Normal Synaptic Morphology, Synaptic Plasticity, and Hippocampus-Dependent Behavior.
- Published in:
- Journal of Neuroscience, 2015, v. 35, n. 49, p. 16018, doi. 10.1523/JNEUROSCI.2009-15.2015
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- Publication type:
- Article
O5‐06‐04: AAV‐CYP46A1 BRAIN DELIVERY MITIGATES ALZHEIMER'S DISEASE: FROM MOUSE MODELS TO NON‐HUMAN PRIMATES.
- Published in:
- Alzheimer's & Dementia: The Journal of the Alzheimer's Association, 2018, v. 14, p. P1658, doi. 10.1016/j.jalz.2018.06.3028
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- Publication type:
- Article
AMYLOID DEPOSITION IS TRIGGERED BY ALZHEIMER'S DISEASE BRAIN HOMOGENATES INOCULATION IN WILD-TYPE MICE WITH FOCAL AD-LIKE PATHOLOGY.
- Published in:
- Alzheimer's & Dementia: The Journal of the Alzheimer's Association, 2016, v. 12, p. P841, doi. 10.1016/j.jalz.2016.06.1721
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- Publication type:
- Article
Gene transfer of both app and ps1 induces hippocampal impairments close to human early phases of Alzheimer’s disease.
- Published in:
- Alzheimer's & Dementia: The Journal of the Alzheimer's Association, 2015, v. 11, n. 7, p. P351, doi. 10.1016/j.jalz.2015.06.233
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- Publication type:
- Article
Improved amyloid pathology contrasting with unexpected memory defects following astrocytic ApoE-e2 overexpression in hippocampus of Alzheimer’s mice.
- Published in:
- Alzheimer's & Dementia: The Journal of the Alzheimer's Association, 2015, v. 11, n. 7, p. P347, doi. 10.1016/j.jalz.2015.06.223
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- Publication type:
- Article
Hippocampal CYP46A1 overexpression is beneficial in rodent models of Alzheimer’s disease.
- Published in:
- Alzheimer's & Dementia: The Journal of the Alzheimer's Association, 2015, v. 11, n. 7, p. P758, doi. 10.1016/j.jalz.2015.06.1691
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- Publication type:
- Article
AAV gene therapy with cholesterol 24-hydroxylase improves Alzheimer's phenotype of tau mouse model
- Published in:
- 2011
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- Publication type:
- Abstract
Enhanced conversion of cholesterol to 24s-hydroxycholesterol decreases amyloid-ß and prevents alzheimer phenotype of app23 mouse model
- Published in:
- 2009
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- Publication type:
- Abstract
Ultramicroscopy as a novel tool to unravel the tropism of AAV gene therapy vectors in the brain.
- Published in:
- Scientific Reports, 2016, p. 28272, doi. 10.1038/srep28272
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- Publication type:
- Article
Inhibiting cholesterol degradation induces neuronal sclerosis and epileptic activity in mouse hippocampus.
- Published in:
- European Journal of Neuroscience, 2015, v. 41, n. 10, p. 1345, doi. 10.1111/ejn.12911
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- Publication type:
- Article
Inhibition of DYRK1A proteolysis modifies its kinase specificity and rescues Alzheimer phenotype in APP/PS1 mice.
- Published in:
- Acta Neuropathologica Communications, 2019, v. 7, n. 1, p. N.PAG, doi. 10.1186/s40478-019-0678-6
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- Publication type:
- Article
Cholesterol Hydroxylating Cytochrome P450 46A1: From Mechanisms of Action to Clinical Applications.
- Published in:
- Frontiers in Aging Neuroscience, 2021, v. 13, p. 1, doi. 10.3389/fnagi.2021.696778
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- Publication type:
- Article
Prevalence of pulmonary tuberculosis among prison inmates: A cross-sectional survey at the Correctional and Detention Facility of Abidjan, Côte d'Ivoire.
- Published in:
- PLoS ONE, 2017, v. 12, n. 7, p. 1, doi. 10.1371/journal.pone.0181995
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- Publication type:
- Article
Hematopoietic Stem Cell Transplantation and Hematopoietic Stem Cell Gene Therapy in X-Linked Adrenoleukodystrophy.
- Published in:
- Brain Pathology, 2010, v. 20, n. 4, p. 857, doi. 10.1111/j.1750-3639.2010.00394.x
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- Publication type:
- Article
Identification of a replication-defective herpes simplex virus for recombinant adeno-associated virus type 2 (rAAV2) particle assembly using stable producer cell lines.
- Published in:
- Journal of Gene Medicine, 2004, v. 6, n. 5, p. 555, doi. 10.1002/jgm.542
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- Publication type:
- Article
CYP46A1, the rate-limiting enzyme for cholesterol degradation, is neuroprotective in Huntington's disease.
- Published in:
- 2016
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- Publication type:
- journal article
CYP46A1 inhibition, brain cholesterol accumulation and neurodegeneration pave the way for Alzheimer's disease.
- Published in:
- 2015
- By:
- Publication type:
- journal article
Prevention and reversal of severe mitochondrial cardiomyopathy by gene therapy in a mouse model of Friedreich's ataxia.
- Published in:
- Nature Medicine, 2014, v. 20, n. 5, p. 542, doi. 10.1038/nm.3510
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- Publication type:
- Article
Abnormal messenger RNA expression and a missense mutation in patients with X-linked adrenoleukodystrophy.
- Published in:
- Human Molecular Genetics, 1993, v. 2, n. 11, p. 1949
- By:
- Publication type:
- Article
Modulation of Brain Cholesterol Metabolism through CYP46A1 Overexpression for Rett Syndrome.
- Published in:
- Pharmaceutics, 2024, v. 16, n. 6, p. 756, doi. 10.3390/pharmaceutics16060756
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- Publication type:
- Article
The cholesterol 24-hydroxylase activates autophagy and decreases mutant huntingtin build-up in a neuroblastoma culture model of Huntington's disease.
- Published in:
- BMC Research Notes, 2020, v. 13, n. 1, p. 1, doi. 10.1186/s13104-020-05053-x
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- Publication type:
- Article
Neuronal Cholesterol Accumulation Induced by Cyp46a1 Down-Regulation in Mouse Hippocampus Disrupts Brain Lipid Homeostasis.
- Published in:
- Frontiers in Molecular Neuroscience, 2017, p. 1, doi. 10.3389/fnmol.2017.00211
- By:
- Publication type:
- Article