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:
Dec. 14, 2021
Filed:
Jun. 16, 2017
Ionis Pharmaceuticals, Inc., Carlsbad, CA (US);
The Johns Hopkins University, Baltimore, MD (US);
Frank Rigo, Carlsbad, CA (US);
C. Frank Bennett, Carlsbad, CA (US);
Constantin Van Outryve D'Ydewalle, Baltimore, MD (US);
Charlotte J. Sumner, Baltimore, MD (US);
Ionis Pharmaceuticals, Inc., Carlsbad, CA (US);
The Johns Hopkins University, Baltimore, MD (US);
Abstract
Certain embodiments are directed to methods and compounds for modulating expression of SMN. In certain embodiments at least two compounds are used: a first compound for inhibiting SMN-NAT and increasing expression of SMN, and a second compound for modulating the splicing of SMN. Such methods and compounds are useful for increasing expression exon 7 containing SMN mRNA in cells and animals.