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US 12,215,320 B2Granted
Compositions and methods for gene editing by targeting transferrin
AssigneesCrispr Therapeutics AG, Bayer HealthCare LLC
InventorsAlan Richard Brooks, Karen Vo
Life of this patent
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Abstract
Provided include compositions, methods, and systems for modulating the expression, function, and/or activity of a target gene, for example a blood-clotting protein such as Factor VIII (FVIII), in a cell by genome editing. Also provided include compositions, methods, and systems for treating a subject having or suspected of having a disorder or health condition, e.g., Hemophilia A, employing ex vivo and/or in vivo genome editing.
Claims15
A system comprising:
- a deoxyribonucleic acid (DNA) endonuclease or nucleic acid encoding the DNA endonuclease;
- a guide RNA (gRNA) comprising a spacer sequence that is complementary to a sequence within intron 1 of an endogenous transferrin gene in a cell;
- and
- a donor template comprising a nucleic acid sequence encoding a protein-of-interest (POI), wherein the POI is Factor VIII (FVIII).
1 more element
Dependencies are inferred from the claim text.