Antisense oligonucleotide therapy for spinocerebellar ataxia type 2

Daniel R. Scoles (), Pratap Meera, Matthew D. Schneider, Sharan Paul, Warunee Dansithong, Karla P. Figueroa, Gene Hung, Frank Rigo, C. Frank Bennett, Thomas S. Otis and Stefan M. Pulst ()
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Daniel R. Scoles: University of Utah
Pratap Meera: University of California Los Angeles
Matthew D. Schneider: University of Utah
Sharan Paul: University of Utah
Warunee Dansithong: University of Utah
Karla P. Figueroa: University of Utah
Gene Hung: Ionis Pharmaceuticals
Frank Rigo: Ionis Pharmaceuticals
C. Frank Bennett: Ionis Pharmaceuticals
Thomas S. Otis: University of California Los Angeles
Stefan M. Pulst: University of Utah

Nature, 2017, vol. 544, issue 7650, 362-366

Abstract: Antisense oligonucleotides against ATXN2 improved motor neuron function and restored firing frequency in cerebellar Purkinje cells in mouse models of spinocerebellar ataxia type 2.

Date: 2017
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DOI: 10.1038/nature22044

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