Engineered meganucleases having specificity for a recognition sequence in the hydroxyacid oxidase 1 gene
AssigneePrecision BioSciences, Inc.
InventorsJames Jefferson Smith, Janel Lape, John Morris
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Abstract
Disclosed are engineered meganucleases that bind and cleave a recognition sequence within a hydroxyacid oxidase 1 (HAO1) gene. The present invention also encompasses methods of using such engineered meganucleases to make genetically-modified cells. Further, the invention encompasses pharmaceutical compositions comprising engineered meganuclease proteins, or nucleic acids encoding engineered meganucleases of the invention, and the use of such compositions for treatment of primary hyperoxaluria type I (PH1).
Claims24
An engineered meganuclease that binds and cleaves a recognition sequence consisting of SEQ ID NO:
- 3 within a hydroxyacid oxidase 1 (HAO1) gene, wherein said engineered meganuclease comprises the amino acid sequence of any one of SEQ ID NOs:
- 5, 6, 7, 10, or 11.
Dependencies are inferred from the claim text.