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CRISPRseek: a bioconductor package to identify target-specific guide RNAs for CRISPR-Cas9 genome-editing systems

PloS one, 2014-09, Vol.9 (9), p.e108424-e108424

2014

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5΄-Vinylphosphonate improves tissue accumulation and efficacy of conjugated siRNAs in vivo

Nucleic acids research, 2017-07, Vol.45 (13), p.7581-7592

2017

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Comparison of partially and fully chemically-modified siRNA in conjugate-mediated delivery in vivo

Nucleic acids research, 2018-03, Vol.46 (5), p.2185-2196

2018

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A synthetic luciferin improves bioluminescence imaging in live mice

Nature methods, 2014-04, Vol.11 (4), p.393-395

2014

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High-resolution proteomic and lipidomic analysis of exosomes and microvesicles from different cell sources

Journal of extracellular vesicles, 2016-01, Vol.5 (1), p.32570-n/a

2016

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Asymmetry in the Assembly of the RNAi Enzyme Complex

Cell, 2003-10, Vol.115 (2), p.199-208

2003

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Recurrent Mutations of Chromatin-Remodeling Genes and Kinase Receptors in Pheochromocytomas and Paragangliomas

Clinical cancer research, 2016-05, Vol.22 (9), p.2301-2310

2016

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Germline mutations in TMEM127 confer susceptibility to pheochromocytoma

Nature genetics, 2010-03, Vol.42 (3), p.229-233

2010

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Five siRNAs Targeting Three SNPs May Provide Therapy for Three-Quarters of Huntington's Disease Patients

Current biology, 2009-05, Vol.19 (9), p.774-778

2009

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Argonaute protein identity and pairing geometry determine cooperativity in mammalian RNA silencing

RNA (Cambridge), 2011-10, Vol.17 (10), p.1858-1869

2011

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Aberrant Rab11-Dependent Trafficking of the Neuronal Glutamate Transporter EAAC1 Causes Oxidative Stress and Cell Death in Huntington's Disease

The Journal of neuroscience, 2010-03, Vol.30 (13), p.4552-4561

2010

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Transvascular Delivery of Hydrophobically Modified siRNAs: Gene Silencing in the Rat Brain upon Disruption of the Blood-Brain Barrier

Molecular therapy, 2018-11, Vol.26 (11), p.2580-2591

2018

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Hydrophobically Modified siRNAs Silence Huntingtin mRNA in Primary Neurons and Mouse Brain

Molecular therapy. Nucleic acids, 2015-12, Vol.4 (12), p.e266-e266, Article e266

2015

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Nuclear Localization of Huntingtin mRNA Is Specific to Cells of Neuronal Origin

Cell reports (Cambridge), 2018-09, Vol.24 (10), p.2553-2560.e5

2018

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Designing siRNA that distinguish between genes that differ by a single nucleotide

PLoS genetics, 2006-09, Vol.2 (9), p.e140

2006

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Docosahexaenoic Acid Conjugation Enhances Distribution and Safety of siRNA upon Local Administration in Mouse Brain

Molecular therapy. Nucleic acids, 2016, Vol.5 (8), p.e344-e344, Article e344

2016

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Huntingtin Expression Stimulates Endosomal-Lysosomal Activity, Endosome Tubulation, and Autophagy

The Journal of neuroscience, 2000-10, Vol.20 (19), p.7268-7278

2000

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Adeno-Associated Virus Neutralizing Antibodies in Large Animals and Their Impact on Brain Intraparenchymal Gene Transfer

Molecular therapy. Methods & clinical development, 2018-12, Vol.11, p.65-72

2018

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Perspective: Tominersen Testing Finds a Way Forward

Journal of Huntington's disease, 2022, Vol.11 (2), p.119-120

Aronin, Neil

2022

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Mutant Huntingtin Impairs Vesicle Formation from Recycling Endosomes by Interfering with Rab11 Activity

Molecular and Cellular Biology, 2009-11, Vol.29 (22), p.6106-6116

2009

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Aggregation of Huntingtin in Neuronal Intranuclear Inclusions and Dystrophic Neurites in Brain

Science (American Association for the Advancement of Science), 1997-09, Vol.277 (5334), p.1990-1993

1997

Volltextzugriff (PDF)

Awake intracerebroventricular delivery and safety assessment of oligonucleotides in a large animal model

Molecular therapy. Methods & clinical development, 2023-12, Vol.31, p.101122-101122, Article 101122

2023

Volltextzugriff (PDF)

Caspase 3-Cleaved N-Terminal Fragments of Wild-Type and Mutant Huntingtin are Present in Normal and Huntington's Disease Brains, Associate with Membranes, and Undergo Calpain-Dependent Proteolysis

Proceedings of the National Academy of Sciences - PNAS, 2001-10, Vol.98 (22), p.12784-12789

2001

Volltextzugriff (PDF)

Excitatory transmission onto AgRP neurons is regulated by cJun NH2-terminal kinase 3 in response to metabolic stress

eLife, 2016-02, Vol.5, p.e10031-e10031

2016

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CRISPRseek: a bioconductor package to identify target-specific guide RNAs for CRISPR-Cas9 genome-editing systems

5΄-Vinylphosphonate improves tissue accumulation and efficacy of conjugated siRNAs in vivo

Comparison of partially and fully chemically-modified siRNA in conjugate-mediated delivery in vivo

A synthetic luciferin improves bioluminescence imaging in live mice

High-resolution proteomic and lipidomic analysis of exosomes and microvesicles from different cell sources

Asymmetry in the Assembly of the RNAi Enzyme Complex

Recurrent Mutations of Chromatin-Remodeling Genes and Kinase Receptors in Pheochromocytomas and Paragangliomas

Germline mutations in TMEM127 confer susceptibility to pheochromocytoma

Five siRNAs Targeting Three SNPs May Provide Therapy for Three-Quarters of Huntington's Disease Patients

Argonaute protein identity and pairing geometry determine cooperativity in mammalian RNA silencing

Aberrant Rab11-Dependent Trafficking of the Neuronal Glutamate Transporter EAAC1 Causes Oxidative Stress and Cell Death in Huntington's Disease

Transvascular Delivery of Hydrophobically Modified siRNAs: Gene Silencing in the Rat Brain upon Disruption of the Blood-Brain Barrier

Hydrophobically Modified siRNAs Silence Huntingtin mRNA in Primary Neurons and Mouse Brain

Nuclear Localization of Huntingtin mRNA Is Specific to Cells of Neuronal Origin

Designing siRNA that distinguish between genes that differ by a single nucleotide

Serum Deprivation of Mesenchymal Stem Cells Improves Exosome Activity and Alters Lipid and Protein Composition

Docosahexaenoic Acid Conjugation Enhances Distribution and Safety of siRNA upon Local Administration in Mouse Brain

Huntingtin Expression Stimulates Endosomal-Lysosomal Activity, Endosome Tubulation, and Autophagy

Adeno-Associated Virus Neutralizing Antibodies in Large Animals and Their Impact on Brain Intraparenchymal Gene Transfer

Perspective: Tominersen Testing Finds a Way Forward

Mutant Huntingtin Impairs Vesicle Formation from Recycling Endosomes by Interfering with Rab11 Activity

Aggregation of Huntingtin in Neuronal Intranuclear Inclusions and Dystrophic Neurites in Brain

Awake intracerebroventricular delivery and safety assessment of oligonucleotides in a large animal model

Caspase 3-Cleaved N-Terminal Fragments of Wild-Type and Mutant Huntingtin are Present in Normal and Huntington's Disease Brains, Associate with Membranes, and Undergo Calpain-Dependent Proteolysis

Excitatory transmission onto AgRP neurons is regulated by cJun NH2-terminal kinase 3 in response to metabolic stress

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