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US 12,595,469 B2Granted
Treatment of retinitis pigmentosa using improved engineered meganucleases
AssigneePrecision BioSciences, Inc.
InventorsVictor Bartsevich, Derek Jantz, James Jefferson Smith, Michael G. Nicholson
Life of this patent
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Abstract
Disclosed are recombinant meganucleases engineered to bind and cleave a recognition sequence present in a mutant RHO P23H allele. The invention further relates to the use of such recombinant meganucleases in a method for treating retinitis pigmentosa, wherein the mutant RHO P23H allele is preferentially targeted, cleaved, and inactivated.
Claims14
An engineered meganuclease that binds and cleaves a recognition sequence consisting of SEQ ID NO:
- 7 in a rhodopsin gene, wherein said engineered meganuclease comprises a first subunit and a second subunit, wherein said first subunit binds to a first recognition half-site of said recognition sequence and comprises a first hypervariable (HVR1) region, wherein said second subunit binds to a second recognition half-site of said recognition sequence and comprises a second hypervariable (HVR2) region, and wherein said engineered meganuclease comprises the amino acid sequence of SEQ ID NO:
- 11.
Dependencies are inferred from the claim text.