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.

Date of Patent:
Jan. 08, 2019

Filed:

May. 16, 2013
Applicants:

Translate Bio Ma, Inc., Lexington, MA (US);

The General Hospital Corporation, Boston, MA (US);

Inventors:

Arthur M. Krieg, Cambridge, MA (US);

Romesh Subramanian, Framingham, MA (US);

James McSwiggen, Arlington, MA (US);

Jeannie T. Lee, Boston, MA (US);

Assignees:

The General Hospital Corporation, Boston, MA (US);

Translate Bio MA, Inc., Lexington, MA (US);

Attorney:
Primary Examiner:
Int. Cl.
CPC ...
C12N 15/113 (2010.01); A61K 31/7125 (2006.01); A61K 31/712 (2006.01); A61K 31/7115 (2006.01); C07H 21/00 (2006.01); C07H 21/02 (2006.01); C07H 21/04 (2006.01); A61K 31/7088 (2006.01); A61K 47/28 (2006.01); A61K 47/64 (2017.01);
U.S. Cl.
CPC ...
C12N 15/113 (2013.01); A61K 31/7088 (2013.01); A61K 31/712 (2013.01); A61K 31/7125 (2013.01); A61K 47/28 (2013.01); A61K 47/64 (2017.08); C07H 21/00 (2013.01); C07H 21/02 (2013.01); C07H 21/04 (2013.01); C12N 2310/11 (2013.01); C12N 2310/315 (2013.01); C12N 2310/3231 (2013.01); C12N 2310/3341 (2013.01); C12N 2310/341 (2013.01);
Abstract

Aspects of the invention provide single stranded oligonucleotides for activating or enhancing expression of hemoglobin genes (HBB, HBD, HBE1, HBG1 or HBG2). Further aspects provide compositions and kits comprising single stranded oligonucleotides for activating or enhancing expression of hemoglobin genes. Methods for modulating expression of hemoglobin genes 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 hemoglobin genes.


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