Methods and compositions for treatment of a genetic condition
First Claim
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1. A CRISPR/Cas nuclease comprising a single guide RNA as shown in SEQ ID NO:
- 151 or 152 that binds to a target site in an enhancer region of a BCL11A gene, wherein the nuclease cleaves and inactivates the BCL11A gene.
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Abstract
Methods and compositions comprising a CRISPR/Cas nuclease that cleaves a BCL11A gene for the modification of the BCL11A gene and for modification of globin expression.
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17 Claims
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1. A CRISPR/Cas nuclease comprising a single guide RNA as shown in SEQ ID NO:
- 151 or 152 that binds to a target site in an enhancer region of a BCL11A gene, wherein the nuclease cleaves and inactivates the BCL11A gene.
- View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17)
Specification