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First published October 15, 2020 Application Aachen patent C – Chemistry & metallurgy

Compositions and methods for modulating Ataxin 3 expression

Espacenet Family 58387449

Abstract

Disclosed are methods for modulating splicing of Ataxin 3 mRNA in an animal with modified oligonucleotides. Such compounds and methods are useful to treat, prevent, or ameliorate spinocerebellar ataxia type 3 (SCA3) in an individual in need thereof.

Inventors 5

  1. Rigo, Frank
  2. Prakash, Thazha P.
  3. van Roon-Mom, Wilhelmina Maria Clasina
  4. Lodewijk J.A. Toonen Aachen author as filed: Toonen, Lodewijk Julius Anton
  5. van Roon-Mom, Wilhemina Maria, Clasina

Inventors are linked to an Aachen author when surname and first name match exactly one author.

Applicants

  • IONIS PHARMACEUTICALS, INC.
  • ACADEMISCH ZIEKENHUIS LEIDEN GBS Leiden

Cited papers 0 in the database

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