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Broad distribution of ataxin 1 silencing in rhesus cerebella for spinocerebellar ataxia type 1 therapy.
- Published in:
- 2015
- By:
- Publication type:
- journal article
A Mouse Model of Classical Late-Infantile Neuronal Ceroid Lipofuscinosis Based on Targeted Disruption of the CLN2 Gene Results in a Loss of Tripeptidyl-Peptidase I Activity and Progressive Neurodegeneration.
- Published in:
- Journal of Neuroscience, 2004, v. 24, n. 41, p. 9117, doi. 10.1523/JNEUROSCI.2729-04.2004
- By:
- Publication type:
- Article
Neurological diseases: Viral vectors for gene delivery to the nervous system.
- Published in:
- Nature Reviews Neuroscience, 2003, v. 4, n. 5, p. 353, doi. 10.1038/nrn1104
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- Publication type:
- Article
Elucidation of transcriptome-wide microRNA binding sites in human cardiac tissues by Ago2 HITS-CLIP.
- Published in:
- Nucleic Acids Research, 2016, v. 44, n. 15, p. 7120, doi. 10.1093/nar/gkw640
- By:
- Publication type:
- Article
Single nucleotide seed modification restores in vivo tolerability of a toxic artificial miRNA sequence in the mouse brain.
- Published in:
- Nucleic Acids Research, 2014, v. 42, n. 21, p. 13315, doi. 10.1093/nar/gku979
- By:
- Publication type:
- Article
siSPOTR: a tool for designing highly specific and potent siRNAs for human and mouse.
- Published in:
- Nucleic Acids Research, 2013, v. 41, n. 1, p. e9, doi. 10.1093/nar/gks797
- By:
- Publication type:
- Article
An orally available, brain penetrant, small molecule lowers huntingtin levels by enhancing pseudoexon inclusion.
- Published in:
- Nature Communications, 2022, v. 13, n. 1, p. 1, doi. 10.1038/s41467-022-28653-6
- By:
- Publication type:
- Article
Doubling down on siRNAs in the brain.
- Published in:
- Nature Biotechnology, 2019, v. 37, n. 8, p. 865, doi. 10.1038/s41587-019-0204-1
- By:
- Publication type:
- Article
Corrigendum: All in the RNA family.
- Published in:
- 2006
- By:
- Publication type:
- Correction Notice
All in the RNA family.
- Published in:
- Nature Biotechnology, 2006, v. 24, n. 8, p. 951, doi. 10.1038/nbt0806-951
- By:
- Publication type:
- Article
siRNA-mediated gene silencing in vitro and in vivo.
- Published in:
- Nature Biotechnology, 2002, v. 20, n. 10, p. 1006, doi. 10.1038/nbt739
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- Publication type:
- Article
The HIV Tat protein transduction domain improves the biodistribution of β-glucuronidase expressed from recombinant viral vectors.
- Published in:
- Nature Biotechnology, 2001, v. 19, n. 7, p. 640, doi. 10.1038/90242
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- Publication type:
- Article
Molecular signatures of disease brain endothelia provide new sites for CNS-directed enzyme therapy.
- Published in:
- Nature Medicine, 2009, v. 15, n. 10, p. 1215, doi. 10.1038/nm.2025
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- Publication type:
- Article
RNAi suppresses polyglutamine-induced neurodegeneration in a model of spinocerebellar ataxia.
- Published in:
- Nature Medicine, 2004, v. 10, n. 8, p. 816, doi. 10.1038/nm1076
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- Publication type:
- Article
Identification of PDGFR as a receptor for AAV-5 transduction.
- Published in:
- Nature Medicine, 2003, v. 9, n. 10, p. 1306, doi. 10.1038/nm929
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- Publication type:
- Article
Changes in the progression of amyloid deposition in APP/PS mice after overexpression of different ApoE isoforms through intraventricular injection of an adeno-associated virus serotype 4
- Published in:
- 2011
- By:
- Publication type:
- Abstract
The role of computed tomography and magnetic resonance imaging in patients with sinusitis with complications.
- Published in:
- 2002
- By:
- Publication type:
- journal article
The Role of Computed Tomography and Magnetic Resonance Imaging in Patients With Sinusitis With Complications.
- Published in:
- Laryngoscope, 2002, v. 112, n. 2, p. 224, doi. 10.1097/00005537-200202000-00005
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- Publication type:
- Article
RNA polymerase III transcribes human microRNAs.
- Published in:
- Nature Structural & Molecular Biology, 2006, v. 13, n. 12, p. 1097, doi. 10.1038/nsmb1167
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- Publication type:
- Article
Mapping PTBP2 binding in human brain identifies SYNGAP1 as a target for therapeutic splice switching.
- Published in:
- Nature Communications, 2023, p. 1, doi. 10.1038/s41467-023-38273-3
- By:
- Publication type:
- Article
DeepRepeat: direct quantification of short tandem repeats on signal data from nanopore sequencing.
- Published in:
- Genome Biology, 2022, v. 23, n. 1, p. 1, doi. 10.1186/s13059-022-02670-6
- By:
- Publication type:
- Article
Sonic Hedgehog Controls the Phenotypic Fate and Therapeutic Efficacy of Grafted Neural Precursor Cells in a Model of Nigrostriatal Neurodegeneration.
- Published in:
- PLoS ONE, 2015, v. 10, n. 9, p. 1, doi. 10.1371/journal.pone.0137136
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- Publication type:
- Article
CLN3 Deficient Cells Display Defects in the ARF1-Cdc42 Pathway and Actin-Dependent Events.
- Published in:
- PLoS ONE, 2014, v. 9, n. 5, p. 1, doi. 10.1371/journal.pone.0096647
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- Publication type:
- Article
RNA Interference-Based Therapy for Spinocerebellar Ataxia Type 7 Retinal Degeneration.
- Published in:
- PLoS ONE, 2014, v. 9, n. 4, p. 1, doi. 10.1371/journal.pone.0095362
- By:
- Publication type:
- Article
MiR-34a Represses Numbl in Murine Neural Progenitor Cells and Antagonizes Neuronal Differentiation.
- Published in:
- PLoS ONE, 2012, v. 7, n. 6, p. 1, doi. 10.1371/journal.pone.0038562
- By:
- Publication type:
- Article
Impact of Human Interleukin-10 on Vector-Induced Inflammation and Early Graft Function in Rat Lung Transplantation.
- Published in:
- American Journal of Respiratory Cell & Molecular Biology, 2003, v. 28, n. 5, p. 616, doi. 10.1165/rcmb.2002-0109OC
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- Publication type:
- Article
Increasing Epithelial Junction Permeability Enhances Gene Transfer to Airway Epithelia In Vivo.
- Published in:
- American Journal of Respiratory Cell & Molecular Biology, 2000, v. 22, n. 2, p. 129, doi. 10.1165/ajrcmb.22.2.3938
- By:
- Publication type:
- Article
Attenuation of Ischemic Inflammatory Response in Mouse Brain Using an Adenoviral Vector to Induce Overexpression of Interleukin-1 Receptor Antagonist.
- Published in:
- Journal of Cerebral Blood Flow & Metabolism, 1998, v. 18, n. 8, p. 840, doi. 10.1097/00004647-199808000-00004
- By:
- Publication type:
- Article
Attenuation of Stroke Size in Rats Using an Adenoviral Vector to Induce Overexpression of Interleukin-1 Receptor Antagonist in Brain.
- Published in:
- Journal of Cerebral Blood Flow & Metabolism, 1995, v. 15, n. 4, p. 547, doi. 10.1038/jcbfm.1995.68
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- Publication type:
- Article
Current prospects for RNA interference-based therapies.
- Published in:
- Nature Reviews Genetics, 2011, v. 12, n. 5, p. 329, doi. 10.1038/nrg2968
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- Publication type:
- Article
CRISPR to the Rescue: Advances in Gene Editing for the FMR1 Gene.
- Published in:
- Brain Sciences (2076-3425), 2019, v. 9, n. 1, p. 17, doi. 10.3390/brainsci9010017
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- Publication type:
- Article
Standard screening methods underreport AAV-mediated transduction and gene editing.
- Published in:
- Nature Communications, 2019, v. 10, n. 1, p. N.PAG, doi. 10.1038/s41467-019-11321-7
- By:
- Publication type:
- Article
The limitations of central nervous systemdirected gene transfer.
- Published in:
- Behavioral & Brain Sciences, 1995, v. 18, n. 1, p. 54, doi. 10.1017/S0140525X0003733X
- By:
- Publication type:
- Article
The Bifunctional microRNA miR-9/miR-9* Regulates REST and CoREST and Is Downregulated in Huntington's Disease.
- Published in:
- Journal of Neuroscience, 2008, v. 28, n. 53, p. 14341, doi. 10.1523/JNEUROSCI.2390-08.2008
- By:
- Publication type:
- Article
Restoring Acid-Sensing Ion Channel-1a in the Amygdala of Knock-Out Mice Rescues Fear Memory But Not Unconditioned Fear Responses.
- Published in:
- Journal of Neuroscience, 2008, v. 28, n. 51, p. 13737, doi. 10.1523/JNEUROSCI.3907-08.2008
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- Publication type:
- Article
A Knock-In Reporter Model of Batten Disease.
- Published in:
- Journal of Neuroscience, 2007, v. 27, n. 37, p. 9826, doi. 10.1523/JNEUROSCI.1710-07.2007
- By:
- Publication type:
- Article
Connecdenn, A Novel DENN Domain-Containing Protein of Neuronal Clathrin-Coated Vesicles Functioning in Synaptic Vesicle Endocytosis.
- Published in:
- Journal of Neuroscience, 2006, v. 26, n. 51, p. 13202, doi. 10.1523/JNEUROSCI.4608-06.2006
- By:
- Publication type:
- Article
Intracranial Delivery of CLN2 Reduces Brain Pathology in a Mouse Model of Classical Late Infantile Neuronal Ceroid Lipofuscinosis.
- Published in:
- Journal of Neuroscience, 2006, v. 26, n. 5, p. 1334, doi. 10.1523/JNEUROSCI.2676-05.2006
- By:
- Publication type:
- Article
Silencing Primary Dystonia: Lentiviral-Mediated RNA Interference Therapy for DYT1 Dystonia.
- Published in:
- Journal of Neuroscience, 2005, v. 25, n. 45, p. 10502, doi. 10.1523/JNEUROSCI.3016-05.2005
- By:
- Publication type:
- Article
Functional Correction of CNS Phenotypes in a Lysosomal Storage Disease Model Using Adeno-Associated Virus Type 4 Vectors.
- Published in:
- Journal of Neuroscience, 2005, v. 25, n. 41, p. 9321, doi. 10.1523/JNEUROSCI.2936-05.2005
- By:
- Publication type:
- Article
CHIP Suppresses Polyglutamine Aggregation and Toxicity In Vitro and In Vivo.
- Published in:
- Journal of Neuroscience, 2005, v. 25, n. 40, p. 9152, doi. 10.1523/JNEUROSCI.3001-05.2005
- By:
- Publication type:
- Article
Transvascular delivery of small interfering RNA to the central nervous system.
- Published in:
- Nature, 2007, v. 448, n. 7149, p. 39, doi. 10.1038/nature05901
- By:
- Publication type:
- Article
Nonallele Specific Silencing of Ataxin-7 Improves Disease Phenotypes in a Mouse Model of SCA7.
- Published in:
- Molecular Therapy, 2014, v. 22, n. 9, p. 1635, doi. 10.1038/mt.2014.108
- By:
- Publication type:
- Article
Broad Therapeutic Benefit After RNAi Expression Vector Delivery to Deep Cerebellar Nuclei: Implications for Spinocerebellar Ataxia Type 1 Therapy.
- Published in:
- Molecular Therapy, 2014, v. 22, n. 3, p. 588, doi. 10.1038/mt.2013.279
- By:
- Publication type:
- Article
Toward RNAi Therapy for the Polyglutamine Disease Machado-Joseph Disease.
- Published in:
- Molecular Therapy, 2013, v. 21, n. 10, p. 1898, doi. 10.1038/mt.2013.144
- By:
- Publication type:
- Article
Silencing Mutant ATXN3 Expression Resolves Molecular Phenotypes in SCA3 Transgenic Mice.
- Published in:
- Molecular Therapy, 2013, v. 21, n. 10, p. 1909, doi. 10.1038/mt.2013.152
- By:
- Publication type:
- Article
Taking a Break From Huntingtin.
- Published in:
- Molecular Therapy, 2012, v. 20, n. 10, p. 1838, doi. 10.1038/mt.2012.191
- By:
- Publication type:
- Article
Sialic Acid Deposition Impairs the Utility of AAV9, but Not Peptide-modified AAVs for Brain Gene Therapy in a Mouse Model of Lysosomal Storage Disease.
- Published in:
- Molecular Therapy, 2012, v. 20, n. 7, p. 1393, doi. 10.1038/mt.2012.100
- By:
- Publication type:
- Article
Targeting Alzheimer’s disease genes with RNA interference: an efficient strategy for silencing mutant alleles.
- Published in:
- Nucleic Acids Research, 2004, v. 32, n. 2, p. 661, doi. 10.1093/nar/gkh208
- By:
- Publication type:
- Article
Deubiquitinase USP7 contributes to the pathogenicity of spinal and bulbar muscular atrophy.
- Published in:
- 2021
- By:
- Publication type:
- journal article