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:
Oct. 17, 2023
Filed:
May. 25, 2018
The Children's Medical Center Corporation, Boston, MA (US);
Daniel Bauer, Cambridge, MA (US);
THE CHILDREN'S MEDICAL CENTER CORPORATION, Cambridge, MA (US);
Abstract
Provided herein are 2'-O-methyl 3′phosphorothioate (MS)-modified synthetic nucleic acid molecules (single guide RNAs (sgRNAs)) for the use with a Cas9 nuclease in combination as a ribonucleoprotein (RNP) complex for an electroporation-based ex vivo targeted gene disruption of the BCL11A erythroid enhancer's +55, +58, or +62, functional regions. Additionally, provided herein are said RNP complexes (Cas9:MS-sgRNA), and compositions comprising the modified synthetic nucleic acid molecules or said RNP complexes, and methods for increasing fetal hemoglobin levels in a cell by disrupting BCL11A expression at the genomic level. Also provided herein are methods and compositions relating to the treatment of hemoglobinopathies by reinduction of fetal hemoglobin levels.