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US 2024/0173434 A1Published application
Compositions and methods for gene editing for hemophilia a
AssigneesCrispr Therapeutics AG, Bayer HealthCare LLC
InventorAlan Richard Brooks
Life of this patent
Estimating the term…
Abstract
Provided include materials and methods for treating Hemophilia A in a subject ex vivo or in vivo. Also provided include materials and methods for knocking in a FVIII-encoding gene in a genome, in particular the locus of albumin gene.
Claims20
1-93. (canceled)
A system comprising:
- a deoxyribonucleic acid (DNA) endonuclease or nucleic acid encoding said DNA endonuclease;
- a guide RNA (gRNA) comprising a spacer sequence that is complementary to a sequence within intron 1 of an albumin gene in a cell, wherein the spacer sequence is a RNA sequence corresponding to the first 20 nucleotides of nucleotide sequence of SEQ ID NO:22;
- and
- a donor template comprising a nucleic acid sequence encoding a Factor VIII (FVIII) protein or functional derivative thereof.
1 more element
A method of editing a genome in a cell, the method comprising:
- providing the following to the cell:
- a guide RNA (gRNA) comprising a spacer sequence that is complementary to a sequence within intron 1 of an albumin gene in a cell, wherein the spacer sequence is a RNA sequence corresponding to the first 20 nucleotides of nucleotide sequence of SEQ ID NO:22;
- a DNA endonuclease or nucleic acid encoding said DNA endonuclease;
- and
- a donor template comprising a nucleic acid sequence encoding a Factor VIII (FVIII) protein or functional derivative.
2 more elements
A method of treating Hemophilia A in a subject, the method comprising:
- providing the following to a cell in the subject:
- a guide RNA (gRNA) comprising a spacer sequence that is complementary to a sequence within intron 1 of an albumin gene in a cell, wherein the spacer sequence is a RNA sequence corresponding to the first 20 nucleotides of nucleotide sequence of SEQ ID NO:22;
- a DNA endonuclease or nucleic acid encoding said DNA endonuclease;
- and
- a donor template comprising a nucleic acid sequence encoding a Factor VIII (FVIII) protein or functional derivative.
2 more elements
Dependencies are inferred from the claim text.