Methods and compositions for prevention or treatment of a disease
First Claim
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1. A fusion molecule comprising (i) an engineered nuclease that binds to a target site within SEQ NOs:
- 3, 4, 5 or 6 of an endogenous gene that produces a microRNA (miRNA), and (ii) a cleavage domain.
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Abstract
Methods and compositions for treatment of a genetic disease are provided.
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Citations
12 Claims
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1. A fusion molecule comprising (i) an engineered nuclease that binds to a target site within SEQ NOs:
- 3, 4, 5 or 6 of an endogenous gene that produces a microRNA (miRNA), and (ii) a cleavage domain.
- View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12)
Specification