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Improved systemic AAV gene therapy with a neurotrophic capsid in Niemann-Pick disease type C1 mice.
- Published in:
- Life Science Alliance, 2021, v. 4, n. 10, p. 1, doi. 10.26508/lsa.202101040
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- Publication type:
- Article
Microarray expression analysis and identification of serum biomarkers for Niemann–Pick disease, type C1.
- Published in:
- Human Molecular Genetics, 2012, v. 21, n. 16, p. 3632, doi. 10.1093/hmg/dds193
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- Publication type:
- Article
Quantitative, Label‐Free Proteomics in the Symptomatic Niemann–Pick, Type C1 Mouse Model Using Standard Flow Liquid Chromatography and Thermal Focusing Electrospray Ionization.
- Published in:
- Proteomics, 2019, v. 19, n. 9, p. N.PAG, doi. 10.1002/pmic.201800432
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- Publication type:
- Article
Efficacy and ototoxicity of different cyclodextrins in Niemann-Pick C disease.
- Published in:
- Annals of Clinical & Translational Neurology, 2016, v. 3, n. 5, p. 366, doi. 10.1002/acn3.306
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- Publication type:
- Article
Chronic Cyclodextrin Treatment of Murine Niemann-Pick C Disease Ameliorates Neuronal Cholesterol and Glycosphingolipid Storage and Disease Progression.
- Published in:
- PLoS ONE, 2009, v. 4, n. 9, p. 1, doi. 10.1371/journal.pone.0006951
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- Publication type:
- Article
Fostering collaborative research for rare genetic disease: the example of niemannpick type C disease.
- Published in:
- Orphanet Journal of Rare Diseases, 2016, v. 11, p. 1, doi. 10.1186/s13023-016-0540-x
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- Publication type:
- Article
Fostering collaborative research for rare genetic disease: the example of niemann-pick type C disease.
- Published in:
- 2016
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- Publication type:
- letter
Transcriptome of HPβCD-treated Niemann-Pick disease type C1 cells highlights GPNMB as a biomarker for therapeutics.
- Published in:
- Human Molecular Genetics, 2021, v. 30, n. 24, p. 2456, doi. 10.1093/hmg/ddab194
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- Publication type:
- Article
Cyclodextrin has conflicting actions on autophagy flux in vivo in brains of normal and Alzheimer model mice.
- Published in:
- Human Molecular Genetics, 2017, v. 26, n. 5, p. 843, doi. 10.1093/hmg/ddx001
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- Publication type:
- Article
Systemic AAV9 gene therapy improves the lifespan of mice with Niemann-Pick disease, type C1.
- Published in:
- Human Molecular Genetics, 2017, v. 26, n. 1, p. 52, doi. 10.1093/hmg/ddw367
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- Publication type:
- Article