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US 2022/0064651 A1Published application
Talen-based and crispr/cas-based gene editing for bruton's tyrosine kinase
OwnerDavid J. RAWLINGS, Courtnee CLOUGH +1inventors
InventorsDavid J. RAWLINGS, Courtnee CLOUGH, Iram F. KHAN
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Abstract
The present disclosure provides improved genome editing compositions and methods for editing a human BTK gene. The disclosure further provides genome edited cells for the prevention, treatment, or amelioration of at least one symptom of X-linked agammaglobulinemia (XLA).
Claims41
A gene editing composition comprising a TALEN that cleaves a target site in the human Bruton's tyrosine kinase (BTK) gene.
A gene editing composition comprising:
- a) a Cas protein or a polynucleotide encoding a Cas protein;
- b) a guide-RNA (gRNA);
- and
- c) a repair template comprising a functional BTK gene or fragment thereof;
- wherein the gene editing system is capable of repairing an endogenous BTK gene in the B cell or inserting a functional BTK gene into the genome of the B cell.
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Dependencies are inferred from the claim text.