Found: 17
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Isogenic GAA-KO Murine Muscle Cell Lines Mimicking Severe Pompe Mutations as Preclinical Models for the Screening of Potential Gene Therapy Strategies.
- Published in:
- International Journal of Molecular Sciences, 2022, v. 23, n. 11, p. 6298, doi. 10.3390/ijms23116298
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- Publication type:
- Article
Overcoming the Challenges Imposed by Humoral Immunity to AAV Vectors to Achieve Safe and Efficient Gene Transfer in Seropositive Patients.
- Published in:
- Frontiers in Immunology, 2022, v. 13, p. 1, doi. 10.3389/fimmu.2022.857276
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- Publication type:
- Article
Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patients.
- Published in:
- Scientific Reports, 2016, p. 36182, doi. 10.1038/srep36182
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- Publication type:
- Article
Deep morphological analysis of muscle biopsies from type III glycogenesis (GSDIII), debranching enzyme deficiency, revealed stereotyped vacuolar myopathy and autophagy impairment.
- Published in:
- Acta Neuropathologica Communications, 2019, v. 7, n. 1, p. N.PAG, doi. 10.1186/s40478-019-0815-2
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- Publication type:
- Article
Human Immune Responses to Adeno-Associated Virus (AAV) Vectors.
- Published in:
- Frontiers in Immunology, 2020, v. 11, p. 1, doi. 10.3389/fimmu.2020.00670
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- Publication type:
- Article
TAAR1 Modulates Cortical Glutamate NMDA Receptor Function.
- Published in:
- Neuropsychopharmacology, 2015, v. 40, n. 9, p. 2217, doi. 10.1038/npp.2015.65
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- Article
Exposure to wild-type AAV drives distinct capsid immunity profiles in humans.
- Published in:
- 2018
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- Publication type:
- journal article
Parkin regulates kainate receptors by interacting with the GluK2 subunit.
- Published in:
- Nature Communications, 2014, v. 5, n. 10, p. 5182, doi. 10.1038/ncomms6182
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- Publication type:
- Article
Let's switch on AAV!
- Published in:
- Science Translational Medicine, 2020, v. 12, n. 529, p. N.PAG, doi. 10.1126/scitranslmed.aba9016
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- Publication type:
- Article
Interfering in Charcot-Marie-Tooth disease 2D.
- Published in:
- Science Translational Medicine, 2019, v. 11, n. 513, p. N.PAG, doi. 10.1126/scitranslmed.aaz3712
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- Publication type:
- Article
Immune-orthogonal orthologs: The solution for genome editing?
- Published in:
- Science Translational Medicine, 2019, v. 11, n. 505, p. N.PAG, doi. 10.1126/scitranslmed.aay7701
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- Publication type:
- Article
Neutralizing antibodies for AAV vectors: The strange case of AAV5.
- Published in:
- Science Translational Medicine, 2019, v. 11, n. 497, p. N.PAG, doi. 10.1126/scitranslmed.aax9567
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- Publication type:
- Article
Transcriptional adaptation: Another reason why your disease model fails you.
- Published in:
- Science Translational Medicine, 2019, v. 11, n. 489, p. N.PAG, doi. 10.1126/scitranslmed.aax1731
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- Publication type:
- Article
Narrative review of glycogen storage disorder type III with a focus on neuromuscular, cardiac and therapeutic aspects.
- Published in:
- Journal of Inherited Metabolic Disease, 2021, v. 44, n. 3, p. 521, doi. 10.1002/jimd.12355
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- Article
Azaspiracid-1 Alters the E-cadherin Pool in Epithelial Cells.
- Published in:
- Toxicological Sciences, 2007, v. 95, n. 2, p. 427
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- Publication type:
- Article
Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration.
- Published in:
- Nature Communications, 2018, v. 9, n. 1, p. 1, doi. 10.1038/s41467-018-06621-3
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- Publication type:
- Article
Exogenous α-Synuclein Decreases Raft Partitioning of Ca<sub>v</sub>2.2 Channels Inducing Dopamine Release.
- Published in:
- Journal of Neuroscience, 2014, v. 34, n. 32, p. 10603, doi. 10.1523/JNEUROSCI.0608-14.2014
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- Publication type:
- Article