Engineered meganucleases having specificity for a recognition sequence in the transthyretin gene
AssigneePrecision BioSciences, Inc.
InventorsCassandra Gorsuch, Jochen Genschel, Janel Lape, Derek Jantz, James Jefferson Smith
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Abstract
The present invention encompasses engineered meganucleases that bind and cleave a recognition sequence within a TTR 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 TTR-associated diseases, such as transthyretin amyloidosis.
Claims17
An engineered meganuclease that binds and cleaves a recognition sequence in a transthyretin (TTR) 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:
- (a) said recognition sequence consists of SEQ ID NO:
- 7 and said engineered meganuclease comprises an amino acid sequence of any one of SEQ ID NOs:
- 11-14;
- or
- (b) said recognition sequence consists of SEQ ID NO:
- 9 and said engineered meganuclease comprises the amino acid sequence of SEQ ID NO:
- 15.
Dependencies are inferred from the claim text.