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 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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12 Claims
- 1. A fusion molecule comprising (i) an engineered nuclease that binds to an endogenous gene that produces a microRNA (miRNA) and (ii) a cleavage domain.
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