BCL11A guide delivery
US11788087B2 · kind B2 · utility
Assignee
Inventor
Key dates
| Filing date | May 25, 2018 |
| Grant date | Oct 17, 2023 |
| Priority date | — |
| Expiry date | Jun 27, 2040 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2310/346
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
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.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.