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Complete Correction of Brain and Spinal Cord Pathology in Metachromatic Leukodystrophy Mice.
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- Frontiers in Molecular Neuroscience, 2021, v. 13, p. 1, doi. 10.3389/fnmol.2021.677895
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- 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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- Article
AAV‐delivered diacylglycerol kinase DGKk achieves long‐term rescue of fragile X syndrome mouse model.
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- EMBO Molecular Medicine, 2022, v. 14, n. 5, p. 1, doi. 10.15252/emmm.202114649
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- Article
Niacin-mediated Tace activation ameliorates CMT neuropathies with focal hypermyelination.
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- EMBO Molecular Medicine, 2016, v. 8, n. 12, p. 1438, doi. 10.15252/emmm.201606349
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- Article
O5‐06‐04: AAV‐CYP46A1 BRAIN DELIVERY MITIGATES ALZHEIMER'S DISEASE: FROM MOUSE MODELS TO NON‐HUMAN PRIMATES.
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- 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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- Article
SCA7 Mouse Cerebellar Pathology Reveals Preferential Downregulation of Key Purkinje Cell-Identity Genes and Shared Disease Signature with SCA1 and SCA2.
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- Journal of Neuroscience, 2021, v. 41, n. 22, p. 4910, doi. 10.1523/JNEUROSCI.1882-20.2021
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- Article
Modulation of Brain Cholesterol Metabolism through CYP46A1 Overexpression for Rett Syndrome.
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- Pharmaceutics, 2024, v. 16, n. 6, p. 756, doi. 10.3390/pharmaceutics16060756
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- Article