Cargando…

Oligonucleotides Targeting DNA Repeats Downregulate Huntingtin Gene Expression in Huntington's Patient-Derived Neural Model System

Huntington's disease (HD) is one of the most common, dominantly inherited neurodegenerative disorders. It affects the striatum, cerebral cortex, and other subcortical structures leading to involuntary movement abnormalities, emotional disturbances, and cognitive impairments. HD is caused by a C...

Descripción completa

Detalles Bibliográficos
Autores principales:	Umek, Tea, Olsson, Thomas, Gissberg, Olof, Saher, Osama, Zaghloul, Eman M., Lundin, Karin E., Wengel, Jesper, Hanse, Eric, Zetterberg, Henrik, Vizlin-Hodzic, Dzeneta, Smith, C. I. Edvard, Zain, Rula
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Mary Ann Liebert, Inc., publishers 2021
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8713517/ https://www.ncbi.nlm.nih.gov/pubmed/34520257 http://dx.doi.org/10.1089/nat.2021.0021

Ejemplares similares

Chemical Modifications and Design Influence the Potency of Huntingtin Anti-Gene Oligonucleotides
por: Saher, Osama, et al.
Publicado: (2023)

CTG repeat-targeting oligonucleotides for down-regulating Huntingtin expression
por: Zaghloul, Eman M., et al.
Publicado: (2017)

Oligonucleotide Binding to Non-B-DNA in MYC
por: Umek, Tea, et al.
Publicado: (2019)

The ability of locked nucleic acid oligonucleotides to pre-structure the double helix: A molecular simulation and binding study
por: Xu, You, et al.
Publicado: (2019)

Sugar and Polymer Excipients Enhance Uptake and Splice-Switching Activity of Peptide-Dendrimer/Lipid/Oligonucleotide Formulations
por: Saher, Osama, et al.
Publicado: (2019)

Oligonucleotide–Palladacycle Conjugates as Splice-Correcting Agents
por: Hande, Madhuri, et al.
Publicado: (2019)

Oligonucleotide Therapies: The Past and the Present
por: Lundin, Karin E., et al.
Publicado: (2015)

Role of Pseudoisocytidine Tautomerization in Triplex-Forming Oligonucleotides: In Silico and in Vitro Studies
por: Hartono, Yossa Dwi, et al.
Publicado: (2017)

Lipophilic Peptide Dendrimers for Delivery of Splice-Switching Oligonucleotides
por: Daralnakhla, Haneen, et al.
Publicado: (2021)

Development of bis-locked nucleic acid (bisLNA) oligonucleotides for efficient invasion of supercoiled duplex DNA
por: Moreno, Pedro M. D., et al.
Publicado: (2013)

Robust Generation of Person-Specific, Synchronously Active Neuronal Networks Using Purely Isogenic Human iPSC-3D Neural Aggregate Cultures
por: Izsak, Julia, et al.
Publicado: (2019)

Targeted Oligonucleotides for Treating Neurodegenerative Tandem Repeat Diseases
por: Zain, Rula, et al.
Publicado: (2019)

TGF-β1 Suppresses Proliferation and Induces Differentiation in Human iPSC Neural in vitro Models
por: Izsak, Julia, et al.
Publicado: (2020)

SAF-A Forms a Complex with BRG1 and Both Components Are Required for RNA Polymerase II Mediated Transcription
por: Vizlin-Hodzic, Dzeneta, et al.
Publicado: (2011)

BET bromodomain inhibitor HMBA synergizes with MEK inhibition in treatment of malignant glioma
por: Funck-Brentano, Elisa, et al.
Publicado: (2020)

Novel Orthogonally Hydrocarbon-Modified Cell-Penetrating Peptide Nanoparticles Mediate Efficient Delivery of Splice-Switching Antisense Oligonucleotides In Vitro and In Vivo
por: Bazaz, Safa, et al.
Publicado: (2021)

Optimizing anti-gene oligonucleotide ‘Zorro-LNA’ for improved strand invasion into duplex DNA
por: Zaghloul, Eman M., et al.
Publicado: (2011)

Next-generation bis-locked nucleic acids with stacking linker and 2′-glycylamino-LNA show enhanced DNA invasion into supercoiled duplexes
por: Geny, Sylvain, et al.
Publicado: (2016)

Accelerated neuronal and synaptic maturation by BrainPhys medium increases Aβ secretion and alters Aβ peptide ratios from iPSC-derived cortical neurons
por: Satir, Tugce Munise, et al.
Publicado: (2020)

Author Correction: Accelerated neuronal and synaptic maturation by BrainPhys medium increases Aβ secretion and alters Aβ peptide ratios from iPSC-derived cortical neurons
por: Satir, Tugce Munise, et al.
Publicado: (2020)

Identification of candidate genetic variants and altered protein expression in neural stem and mature neural cells support altered microtubule function to be an essential component in bipolar disorder
por: Truvé, Katarina, et al.
Publicado: (2020)

Huntingtin Aggregates in the Olfactory Bulb in Huntington’s Disease
por: Highet, Blake, et al.
Publicado: (2020)

Cationic oligonucleotide derivatives and conjugates: A favorable approach for enhanced DNA and RNA targeting oligonucleotides
por: Danielsen, Mathias B, et al.
Publicado: (2021)

Emerging Therapies for Huntington’s Disease – Focus on N-Terminal Huntingtin and Huntingtin Exon 1
por: van der Bent, M Leontien, et al.
Publicado: (2022)

Allele-Specific Suppression of Mutant Huntingtin Using Antisense Oligonucleotides: Providing a Therapeutic Option for All Huntington Disease Patients
por: Skotte, Niels H., et al.
Publicado: (2014)

Phosphorylation of huntingtin at residue T3 is decreased in Huntington’s disease and modulates mutant huntingtin protein conformation
por: Cariulo, Cristina, et al.
Publicado: (2017)

A pathogenic proteolysis–resistant huntingtin isoform induced by an antisense oligonucleotide maintains huntingtin function
por: Kim, Hyeongju, et al.
Publicado: (2022)

Making (anti-) sense out of huntingtin levels in Huntington disease
por: Evers, Melvin M, et al.
Publicado: (2015)

Huntington's disease cerebrospinal fluid seeds aggregation of mutant huntingtin
por: Tan, Z, et al.
Publicado: (2015)

Salivary levels of total huntingtin are elevated in Huntington’s disease patients
por: Corey-Bloom, Jody, et al.
Publicado: (2018)

Recent Trends in Detection of Huntingtin and Preclinical Models of Huntington's Disease
por: Mantha, Neelima, et al.
Publicado: (2014)

Cyclodextrin-Based Nanoparticles for Delivery of Antisense Oligonucleotides Targeting Huntingtin
por: Mendonça, Monique C. P., et al.
Publicado: (2023)

2′-O-(N-(Aminoethyl)carbamoyl)methyl Modification Allows for Lower Phosphorothioate Content in Splice-Switching Oligonucleotides with Retained Activity
por: Honcharenko, Dmytro, et al.
Publicado: (2022)

LNA effects on DNA binding and conformation: from single strand to duplex and triplex structures
por: Pabon-Martinez, Y. Vladimir, et al.
Publicado: (2017)

Huntington’s Disease: From Mutant Huntingtin Protein to Neurotrophic Factor Therapy
por: Sari, Youssef
Publicado: (2011)

Embryonic Mutant Huntingtin Aggregate Formation in Mouse Models of Huntington’s Disease
por: Osmand, Alexander P., et al.
Publicado: (2016)

A Fresh Look at Huntingtin mRNA Processing in Huntington’s Disease
por: Romo, Lindsay, et al.
Publicado: (2018)

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

Polyglutamine expansion affects huntingtin conformation in multiple Huntington’s disease models
por: Daldin, Manuel, et al.
Publicado: (2017)

Huntington’s Disease Protein Huntingtin Associates with its own mRNA
por: Culver, Brady P., et al.
Publicado: (2016)

Cannot write session to /tmp/vufind_sessions/sess_eltejl3kabv1qdvg6kdkb1jmqs