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:
Aug. 29, 2023

Filed:

Aug. 17, 2021
Applicant:

The Children's Medical Center Corporation, Boston, MA (US);

Inventors:

Michael Milsom, Dossenheim, DE;

David A. Williams, Dover, MA (US);

Richard Gregory, Brookline, MA (US);

Assignee:
Attorneys:
Primary Examiner:
Int. Cl.
CPC ...
C07H 21/04 (2006.01); C12N 15/113 (2010.01); A61K 35/28 (2015.01); C12N 7/00 (2006.01);
U.S. Cl.
CPC ...
C12N 15/1135 (2013.01); A61K 35/28 (2013.01); C12N 7/00 (2013.01); C12N 15/113 (2013.01); C12N 2310/141 (2013.01); C12N 2310/3519 (2013.01); C12N 2310/531 (2013.01); C12N 2740/10032 (2013.01); C12N 2740/10043 (2013.01);
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.


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