Invention Grant
- Patent Title: Compositions and methods to treating hemoglobinopathies
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Application No.: US16366900Application Date: 2019-03-27
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Publication No.: US10662429B2Publication Date: 2020-05-26
- Inventor: Michael Milsom , David A. Williams , Richard Gregory
- Applicant: THE CHILDREN'S MEDICAL CENTER CORPORATION
- Applicant Address: US MA Boston
- Assignee: THE CHILDREN'S MEDICAL CENTER CORPORATION
- Current Assignee: THE CHILDREN'S MEDICAL CENTER CORPORATION
- Current Assignee Address: US MA Boston
- Agency: Nixon Peabody LLP
- Main IPC: C07H21/04
- IPC: C07H21/04 ; C12N15/113 ; A61K35/28 ; C12N7/00

Abstract:
Embodiments herein provide specially designed synthetic BCL11A-targeting microRNAs for RNA polymerase II expression, and methods of use to treat hemoglobinopathies such as sickle cell disease or thalassemia by increasing the expression levels of fetal hemoglobin levels.
Public/Granted literature
- US20190316136A1 COMPOSITIONS AND METHODS TO TREATING HEMOGLOBINOPATHIES Public/Granted day:2019-10-17
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