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:
Nov. 17, 2020
Filed:
Jan. 16, 2018
Translate Bio Ma, Inc., Cambridge, MA (US);
The General Hospital Corporation, Boston, MA (US);
Arthur M. Krieg, Cambridge, MA (US);
Romesh Subramanian, Framingham, MA (US);
James McSwiggen, Arlington, MA (US);
Jeannie T. Lee, Boston, MA (US);
Translate Bio MA, Inc., Lexington, MA (US);
Abstract
Aspects of the invention provide single stranded oligonucleotides for activating or enhancing expression of SMN1 or SMN2. Further aspects provide compositions and kits comprising single stranded oligonucleotides for activating or enhancing expression of SMN1 or SMN2 that comprises exon 7. Methods for modulating expression of SMN1 or SMN2 using the single stranded oligonucleotides are also provided. Further aspects of the invention provide methods for selecting a candidate oligonucleotide for activating or enhancing expression of SMN1 or SMN2.