Cargando…

Removal of the Polyglutamine Repeat of Ataxin-3 by Redirecting pre-mRNA Processing

Spinocerebellar ataxia type 3 (SCA3) is a devastating neurodegenerative disease for which there is currently no cure, nor effective treatment strategy. One of nine polyglutamine disorders known to date, SCA3 is clinically heterogeneous and the main feature is progressive ataxia, which in turn affect...

Descripción completa

Detalles Bibliográficos
Autores principales:	McIntosh, Craig S., Aung-Htut, May Thandar, Fletcher, Sue, Wilton, Steve D.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	MDPI 2019
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6862616/ https://www.ncbi.nlm.nih.gov/pubmed/31683630 http://dx.doi.org/10.3390/ijms20215434

Ejemplares similares

Polyglutamine Ataxias: Our Current Molecular Understanding and What the Future Holds for Antisense Therapies
por: McIntosh, Craig S., et al.
Publicado: (2021)

Induction of cryptic pre-mRNA splice-switching by antisense oligonucleotides
por: Ham, Kristin A., et al.
Publicado: (2021)

In Vitro Validation of Phosphorodiamidate Morpholino Oligomers
por: Aung-Htut, May T., et al.
Publicado: (2019)

Correction to: Neurodegenerative diseases: a hotbed for splicing defects and the potential therapies
por: Li, Dunhui, et al.
Publicado: (2021)

Neurodegenerative diseases: a hotbed for splicing defects and the potential therapies
por: Li, Dunhui, et al.
Publicado: (2021)

Nonsequential Splicing Events Alter Antisense-Mediated Exon Skipping Outcome in COL7A1
por: Ham, Kristin A., et al.
Publicado: (2020)

Splice correction therapies for familial hypercholesterolemic patients with low-density lipoprotein receptor mutations
por: McIntosh, Craig S., et al.
Publicado: (2021)

Polyglutamine Tract Expansion Increases S-Nitrosylation of Huntingtin and Ataxin-1
por: Ni, Chun-Lun, et al.
Publicado: (2016)

Consequences of Making the Inactive Active Through Changes in Antisense Oligonucleotide Chemistries
por: Zaw, Khine, et al.
Publicado: (2019)

Systematic Approach to Developing Splice Modulating Antisense Oligonucleotides
por: Aung-Htut, May T., et al.
Publicado: (2019)

Splice modulating antisense oligonucleotides restore some acid-alpha-glucosidase activity in cells derived from patients with late-onset Pompe disease
por: Aung-Htut, May Thandar, et al.
Publicado: (2020)

Polyglutamine Expansion Accelerates the Dynamics of Ataxin-1 and Does Not Result in Aggregate Formation
por: Krol, Hilde A., et al.
Publicado: (2008)

Ataxin-2 intermediate-length polyglutamine expansions are associated with increased risk for ALS
por: Elden, Andrew C., et al.
Publicado: (2010)

Autophagy mediates SUMO-induced degradation of a polyglutamine protein ataxin-3
por: Hwang, Soo Pyung, et al.
Publicado: (2017)

PML clastosomes prevent nuclear accumulation of mutant ataxin-7 and other polyglutamine proteins
por: Janer, Alexandre, et al.
Publicado: (2006)

Large-Scale Screen for Modifiers of Ataxin-3-Derived Polyglutamine-Induced Toxicity in Drosophila
por: Voßfeldt, Hannes, et al.
Publicado: (2012)

Deletion of Dystrophin In-Frame Exon 5 Leads to a Severe Phenotype: Guidance for Exon Skipping Strategies
por: Toh, Zhi Yon Charles, et al.
Publicado: (2016)

A Splice Intervention Therapy for Autosomal Recessive Juvenile Parkinson’s Disease Arising from Parkin Mutations
por: Li, Dunhui, et al.
Publicado: (2020)

A Major Role for Side-Chain Polyglutamine Hydrogen Bonding in Irreversible Ataxin-3 Aggregation
por: Natalello, Antonino, et al.
Publicado: (2011)

p62/Sequestosome 1 Regulates Aggresome Formation of Pathogenic Ataxin-3 with Expanded Polyglutamine
por: Zhou, Liang, et al.
Publicado: (2014)

The Relationship between Aggregation and Toxicity of Polyglutamine-Containing Ataxin-3 in the Intracellular Environment of Escherichia coli
por: Invernizzi, Gaetano, et al.
Publicado: (2012)

A tale of a tail: Structural insights into the conformational properties of the polyglutamine protein ataxin-3
por: Scarff, Charlotte A., et al.
Publicado: (2013)

Urine levels of the polyglutamine ataxin-3 protein are elevated in patients with spinocerebellar ataxia type 3
por: Koike, Yuka, et al.
Publicado: (2021)

Polyglutamine Repeats in Viruses
por: Schein, Catherine H.
Publicado: (2018)

Induced alternative splicing an opportunity to study PCSK9 protein isoforms at physiologically relevant concentrations
por: Cale, Jessica M., et al.
Publicado: (2023)

Reduction of integrin alpha 4 activity through splice modulating antisense oligonucleotides
por: Aung-Htut, May T., et al.
Publicado: (2019)

Novel Mutations Found in Individuals with Adult-Onset Pompe Disease
por: Aung-Htut, May T., et al.
Publicado: (2020)

The ataxia protein sacsin is a functional co-chaperone that protects against polyglutamine-expanded ataxin-1
por: Parfitt, David A., et al.
Publicado: (2009)

Cross-species genetic screens identify transglutaminase 5 as a regulator of polyglutamine-expanded ataxin-1
por: Lee, Won-Seok, et al.
Publicado: (2022)

Conserved Pbp1/Ataxin-2 regulates retrotransposon activity and connects polyglutamine expansion-driven protein aggregation to lifespan-controlling rDNA repeats
por: Ostrowski, Lauren A., et al.
Publicado: (2018)

Splice Isoforms of the Polyglutamine Disease Protein Ataxin-3 Exhibit Similar Enzymatic yet Different Aggregation Properties
por: Harris, Ginny Marie, et al.
Publicado: (2010)

The polyglutamine protein ataxin-3 enables normal growth under heat shock conditions in the methylotrophic yeast Pichia pastoris
por: Bonanomi, Marcella, et al.
Publicado: (2017)

Identification of a novel site of interaction between ataxin-3 and the amyloid aggregation inhibitor polyglutamine binding peptide 1
por: Knight, Patrick D, et al.
Publicado: (2017)

Ubiquitin-interacting motifs of ataxin-3 regulate its polyglutamine toxicity through Hsc70-4-dependent aggregation
por: Johnson, Sean L, et al.
Publicado: (2020)

Antisense Oligonucleotide-Mediated Removal of the Polyglutamine Repeat in Spinocerebellar Ataxia Type 3 Mice
por: Toonen, Lodewijk J.A., et al.
Publicado: (2017)

Aggregation of polyglutamine-expanded ataxin-3 sequesters its specific interacting partners into inclusions: Implication in a loss-of-function pathology
por: Yang, Hui, et al.
Publicado: (2014)

Polyglutamine-expanded ataxin3 alter specific gene expressions through changing DNA methylation status in SCA3/MJD
por: Ding, Dongxue, et al.
Publicado: (2020)

Model Organisms Reveal Insight into Human Neurodegenerative Disease: Ataxin-2 Intermediate-Length Polyglutamine Expansions Are a Risk Factor for ALS
por: Bonini, Nancy M., et al.
Publicado: (2011)

Publisher Correction: The polyglutamine protein ataxin-3 enables normal growth under heat shock conditions in the methylotrophic yeast Pichia pastoris
por: Bonanomi, Marcella, et al.
Publicado: (2018)

DNAzyme Cleavage of CAG Repeat RNA in Polyglutamine Diseases
por: Zhang, Nan, et al.
Publicado: (2021)

Cannot write session to /tmp/vufind_sessions/sess_qmv4qm99s7udmu3kqgl98qltea