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Antisense Oligonucleotide-Mediated Removal of the Polyglutamine Repeat in Spinocerebellar Ataxia Type 3 Mice

Toonen, L.J.A.
Rigo, F.
Attikum, H. van
Roon-Mom, W.M.C. van

Publication date

September 2017

DOI

10.1016/j.omtn.2017.06.019

Publisher

Elsevier BV

Abstract

Functional Genomics of Muscle, Nerve and Brain DisordersGenome Instability and Cance

Extracted data

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Rubinsztein, David

January 2010

Spinocerebellar ataxia type 3 is a neurodegenerative disorder caused by the expansion of the polyglu...

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Mason, Amanda Gayle

January 2014

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Fabian Stahl
Ina Schmitt
Philip Denner
Laura de Boni
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Peter Breuer

September 2023

Abstract The spinocerebellar ataxias (SCA) comprise a group of inherited neurodegenerative diseases....

Identification of genes that modify ataxin-1-induced neurodegeneration

Fernandez-Funez, Pedro
Nino-Rosales, Maria Laura
de Gouyon, Beatrice
She, Wei-Chi
Luchak, James
Martinez, Pedro
Turiegano, Enrique
Benito, Jonathan
Capovilla, Maria
Skinner, Pamela
McCall, Alanna
Canal, Inmaculada
Orr, Harry
Zoghbi, Huda
Botas, Juan

November 2000

International audienceA growing number of human neurodegenerative diseases result from the expansion...

Polyglutamine ataxias: Our current molecular understanding and what the future holds for antisense therapies

McIntosh, C.S.
Li, D.
Wilton, S.D.
Aung-Htut, M.T.

January 2021

Polyglutamine (polyQ) ataxias are a heterogenous group of neurological disorders all caused by an ex...