The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.
The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.
Patent No.:
Date of Patent:
Jan. 14, 2020
Filed:
Sep. 23, 2016
Applicants:
Ionis Pharmaceuticals, Inc., Carlsbad, CA (US);
Academisch Ziekenhuis Leiden H.o.d.n. Lumc, Leiden, NL;
Inventors:
Frank Rigo, Carlsbad, CA (US);
Thazha P. Prakash, Carlsbad, CA (US);
Wilhelmina Maria Clasina Van Roon-Mom, Leiden, NL;
Lodewijk Julius Anton Toonen, Leiden, NL;
Assignees:
Ionis Pharmaceuticals, Inc., Carlsbad, CA (US);
Academisch Ziekenhuis Leiden, Leiden, NL;
Attorney:
Primary Examiner:
Int. Cl.
CPC ...
C12N 15/113 (2010.01); A61K 31/712 (2006.01); C12N 9/48 (2006.01);
U.S. Cl.
CPC ...
C12N 15/113 (2013.01); A61K 31/712 (2013.01); C12N 9/485 (2013.01); C12Y 304/19012 (2013.01); C12N 2310/11 (2013.01); C12N 2310/321 (2013.01); C12N 2310/3231 (2013.01); C12N 2310/3341 (2013.01); C12N 2310/3525 (2013.01);
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.