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US 2026/0234577 A1Published application
Integrase variants for gene insertion in human cell
AssigneeApplied Stemcell, Inc.
InventorsYanru Ruby TSAI, Alfonso FARRUGGIO
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Abstract
Disclosed herein is a polypeptide comprising a variant of phiC31 integrase linked to a gRNA binding domain wherein the polypeptide recognizes a specific sequence in human genome. Also disclosed are compositions containing the polypeptide or the nucleic acid encoding the polypeptide, and methods of using the compositions, for inserting a transgene to the locus in human genome that contains the specific sequence.
Claims17
A fusion polypeptide comprising a variant of phiC31 integrase linked to a gRNA binding domain, wherein
- (i) the gRNA binding domain is capable of binding to a guide RNA;
- and
- (ii) the fusion polypeptide is capable of integrating a donor DNA sequence to locus of a genomic sequence of SEQ ID NO:
- 2 in a human cell.
1 more element
Dependencies are inferred from the claim text.