Invention Grant
- Patent Title: Compositions and methods for modulating hemoglobin gene family expression
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Application No.: US14401201Application Date: 2013-05-16
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Publication No.: US10174315B2Publication Date: 2019-01-08
- Inventor: Arthur M. Krieg , Romesh Subramanian , James McSwiggen , Jeannie T. Lee
- Applicant: Translate Bio MA, Inc. , The General Hospital Corporation
- Applicant Address: US MA Boston US MA Lexington
- Assignee: The General Hospital Corporation,Translate Bio MA, Inc.
- Current Assignee: The General Hospital Corporation,Translate Bio MA, Inc.
- Current Assignee Address: US MA Boston US MA Lexington
- Agency: Wolf, Greenfield & Sacks, P.C.
- International Application: PCT/US2013/041382 WO 20130516
- International Announcement: WO2013/173599 WO 20131121
- Main IPC: C12N15/113
- IPC: C12N15/113 ; A61K31/7125 ; A61K31/712 ; A61K31/7115 ; C07H21/00 ; C07H21/02 ; C07H21/04 ; A61K31/7088 ; A61K47/28 ; A61K47/64
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.
Public/Granted literature
- US20150133528A1 COMPOSITIONS AND METHODS FOR MODULATING HEMOGLOBIN GENE FAMILY EXPRESSION Public/Granted day:2015-05-14
Information query
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