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

Spinocerebellar ataxia type 3 (SCA3) is a currently incurable neurodegenerative disorder caused by a CAG triplet expansion in exon 10 of the ATXN3 gene. The resultant expanded polyglutamine stretch in the mutant ataxin-3 protein causes a gain of toxic function, which eventually leads to neurodegener...

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Bibliographic Details
Main Authors: Lodewijk J.A. Toonen (Author), Frank Rigo (Author), Haico van Attikum (Author), Willeke M.C. van Roon-Mom (Author)
Format: Book
Published: Elsevier, 2017-09-01T00:00:00Z.
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3rd Floor Main Library

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Call Number: A1234.567
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