Cargando…

Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington’s disease models

Small interfering RNAs are a new class of drugs, exhibiting sequence-driven, potent, and sustained silencing of gene expression in vivo. We recently demonstrated that siRNA chemical architectures can be optimized to provide efficient delivery to the CNS, enabling development of CNS-targeted therapeu...

Descripción completa

Detalles Bibliográficos
Autores principales:	Conroy, Faith, Miller, Rachael, Alterman, Julia F., Hassler, Matthew R., Echeverria, Dimas, Godinho, Bruno M. D. C., Knox, Emily G., Sapp, Ellen, Sousa, Jaquelyn, Yamada, Ken, Mahmood, Farah, Boudi, Adel, Kegel-Gleason, Kimberly, DiFiglia, Marian, Aronin, Neil, Khvorova, Anastasia, Pfister, Edith L.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Nature Publishing Group UK 2022
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9530163/ https://www.ncbi.nlm.nih.gov/pubmed/36192390 http://dx.doi.org/10.1038/s41467-022-33061-x

Ejemplares similares

Structurally constrained phosphonate internucleotide linkage impacts oligonucleotide-enzyme interaction, and modulates siRNA activity and allele specificity
por: Yamada, Ken, et al.
Publicado: (2021)

Di-valent siRNA-mediated silencing of MSH3 blocks somatic repeat expansion in mouse models of Huntington’s disease
por: O’Reilly, Daniel, et al.
Publicado: (2023)

Hydrophobically Modified siRNAs Silence Huntingtin mRNA in Primary Neurons and Mouse Brain
por: Alterman, Julia F, et al.
Publicado: (2015)

2′-O-Methyl at 20-mer Guide Strand 3′ Termini May Negatively Affect Target Silencing Activity of Fully Chemically Modified siRNA
por: Davis, Sarah M., et al.
Publicado: (2020)

Rac1 Activity Is Modulated by Huntingtin and Dysregulated in Models of Huntington’s Disease
por: Tousley, Adelaide, et al.
Publicado: (2019)

A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system
por: Alterman, Julia F., et al.
Publicado: (2019)

5΄-Vinylphosphonate improves tissue accumulation and efficacy of conjugated siRNAs in vivo
por: Haraszti, Reka A., et al.
Publicado: (2017)

Early whole-body mutant huntingtin lowering averts changes in proteins and lipids important for synapse function and white matter maintenance in the LacQ140 mouse model
por: Shing, Kai, et al.
Publicado: (2023)

Disposition of Proteins and Lipids in Synaptic Membrane Compartments Is Altered in Q175/Q7 Huntington’s Disease Mouse Striatum
por: Iuliano, Maria, et al.
Publicado: (2021)

Docosahexaenoic Acid Conjugation Enhances Distribution and Safety of siRNA upon Local Administration in Mouse Brain
por: Nikan, Mehran, et al.
Publicado: (2016)

Extended Nucleic Acid (exNA): A Novel, Biologically Compatible Backbone that Significantly Enhances Oligonucleotide Efficacy in vivo
por: Hariharan, Vignesh Narayan, et al.
Publicado: (2023)

Single-Stranded Phosphorothioated Regions Enhance Cellular Uptake of Cholesterol-Conjugated siRNA but Not Silencing Efficacy
por: Ly, Socheata, et al.
Publicado: (2020)

Comparative route of administration studies using therapeutic siRNAs show widespread gene modulation in Dorset sheep
por: Ferguson, Chantal M., et al.
Publicado: (2021)

Huntingtin associates with the actin cytoskeleton and α-actinin isoforms to influence stimulus dependent morphology changes
por: Tousley, Adelaide, et al.
Publicado: (2019)

PK-modifying anchors significantly alter clearance kinetics, tissue distribution, and efficacy of therapeutics siRNAs
por: Godinho, Bruno M.D.C., et al.
Publicado: (2022)

Cell Type Impacts Accessibility of mRNA to Silencing by RNA Interference
por: Ferguson, Chantal M., et al.
Publicado: (2020)

Chemical optimization of siRNA for safe and efficient silencing of placental sFLT1
por: Davis, Sarah M., et al.
Publicado: (2022)

Autophagy Activation by Transcription Factor EB (TFEB) in Striatum of HD(Q175/Q7) Mice
por: Vodicka, Petr, et al.
Publicado: (2016)

Diverse lipid conjugates for functional extra-hepatic siRNA delivery in vivo
por: Biscans, Annabelle, et al.
Publicado: (2019)

Extended Nucleic Acid (exNA): A Novel, Biologically Compatible Backbone that Significantly Enhances Oligonucleotide Efficacy in vivo.
por: Yamada, Ken, et al.
Publicado: (2023)

Impaired XK recycling for importing manganese underlies striatal vulnerability in Huntington's disease
por: Chhetri, Gaurav, et al.
Publicado: (2022)

Comparison of partially and fully chemically-modified siRNA in conjugate-mediated delivery in vivo
por: Hassler, Matthew R, et al.
Publicado: (2018)

Serum Deprivation of Mesenchymal Stem Cells Improves Exosome Activity and Alters Lipid and Protein Composition
por: Haraszti, Reka Agnes, et al.
Publicado: (2019)

Hydrophobicity drives the systemic distribution of lipid-conjugated siRNAs via lipid transport pathways
por: Osborn, Maire F, et al.
Publicado: (2019)

Guanabenz (Wytensin™) selectively enhances uptake and efficacy of hydrophobically modified siRNAs
por: Osborn, Maire F., et al.
Publicado: (2015)

Visualization of self-delivering hydrophobically modified siRNA cellular internalization
por: Ly, Socheata, et al.
Publicado: (2017)

Glucose transporter 3 is a rab11-dependent trafficking cargo and its transport to the cell surface is reduced in neurons of CAG140 Huntington’s disease mice
por: McClory, Hollis, et al.
Publicado: (2014)

Hippo Signaling Pathway Dysregulation in Human Huntington’s Disease Brain and Neuronal Stem Cells
por: Mueller, Kaly A., et al.
Publicado: (2018)

The COOH-terminal domain of huntingtin interacts with RhoGEF kalirin and modulates cell survival
por: McClory, Hollis, et al.
Publicado: (2018)

Assessing average somatic CAG repeat instability at the protein level
por: Aviolat, Hubert, et al.
Publicado: (2019)

High-resolution proteomic and lipidomic analysis of exosomes and microvesicles from different cell sources
por: Haraszti, Reka A., et al.
Publicado: (2016)

Taming the Huntington’s Disease Proteome: What Have We Learned?
por: Seeley, Connor, et al.
Publicado: (2021)

Induced Pluripotent Stem Cells in Huntington’s Disease Research: Progress and Opportunity
por: Tousley, Adelaide, et al.
Publicado: (2016)

Heavily and fully modified RNAs guide efficient SpyCas9-mediated genome editing
por: Mir, Aamir, et al.
Publicado: (2018)

DIPG-17. TREATMENT OF DIFFUSE INTRINSIC PONTINE GLIOMA USING ALLELE-SPECIFIC SIRNA TARGETING H3.3 K27M MUTANT HISTONE
por: Wentworth, Simon, et al.
Publicado: (2023)

Huntingtin cleavage product A forms in neurons and is reduced by gamma-secretase inhibitors
por: Kegel, Kimberly B, et al.
Publicado: (2010)

Safe and Efficient Silencing with a Pol II, but Not a Pol lII, Promoter Expressing an Artificial miRNA Targeting Human Huntingtin
por: Pfister, Edith L., et al.
Publicado: (2017)

Kalirin Interacts with TRAPP and Regulates Rab11 and Endosomal Recycling
por: Wang, Xiaolong, et al.
Publicado: (2020)

Htt(Q111/+) Huntington’s Disease Knock-in Mice Exhibit Brain Region-Specific Morphological Changes and Synaptic Dysfunction
por: Kovalenko, Marina, et al.
Publicado: (2018)

RNAi Modulation of Placental sFLT1 for the Treatment of Preeclampsia.
por: Turanov, Anton A., et al.
Publicado: (2018)

Cannot write session to /tmp/vufind_sessions/sess_8qa7893qgjka1cqlvsajagce3a