Targeted integration into stem cells
First Claim
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1. An isolated pluripotent or multipotent stem cell comprising a transgene, wherein the transgene is integrated into an endogenous PITX3 or Factor IX gene of the stem cell using a pair of zinc finger nucleases that bind to and cleave the endogenous PITX3 or Factor IX gene, wherein the pair of zinc finger nucleases bind to paired target sites of SEQ ID NO:
- 47 and SEQ ID NO;
48 of the PITX3 gene, or paired target sites of SEQ ID NO;
49 and SEQ ID NO;
50 of the PITX3 gene, or to paired target sites of SEQ ID NO;
51 and SEQ ID NO;
56 of the Factor IX gene, and further wherein the pair of zinc finger nucleases cleaves the PITX3 or Factor IX gene within or between the paired target sites such that the transgene is integrated into the PITX3 or Factor IX gene.
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Abstract
Disclosed herein are methods and compositions for targeted integration of sequences of interest such as lineage-specific or cell fate reporter constructs or protein encoding sequences into stem cells.
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Citations
7 Claims
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1. An isolated pluripotent or multipotent stem cell comprising a transgene, wherein the transgene is integrated into an endogenous PITX3 or Factor IX gene of the stem cell using a pair of zinc finger nucleases that bind to and cleave the endogenous PITX3 or Factor IX gene, wherein the pair of zinc finger nucleases bind to paired target sites of SEQ ID NO:
- 47 and SEQ ID NO;
48 of the PITX3 gene, or paired target sites of SEQ ID NO;
49 and SEQ ID NO;
50 of the PITX3 gene, or to paired target sites of SEQ ID NO;
51 and SEQ ID NO;
56 of the Factor IX gene, and further wherein the pair of zinc finger nucleases cleaves the PITX3 or Factor IX gene within or between the paired target sites such that the transgene is integrated into the PITX3 or Factor IX gene. - View Dependent Claims (2, 3, 4, 5, 6, 7)
- 47 and SEQ ID NO;
Specification