Gene editing methods for treating alpha-1 antitrypsin (AAT) deficiency
AssigneePrecision BioSciences, Inc.
InventorsJames Jefferson Smith, John Morris, Janel Lape, Cassandra Gorsuch, Paige Scarlett Nemec, Jason Richard Harris, Wendy Shoop
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Abstract
Disclosed are engineered meganucleases that bind and cleave a recognition sequence within a serine peptidase inhibitor, Clade A, Member 1 (SERPINA1) gene, which encodes alpha-1 antitrypsin (AAT). Further disclosed are donor polynucleotides that encode functional AAT proteins. The present disclosure also encompasses methods of using such engineered meganucleases and donor polynucleotides to make genetically-modified cells and use of such compositions for treatment of AAT deficiency.
Claims4
An engineered meganuclease that binds and cleaves a recognition sequence comprising SEQ ID NO:
- 9 within a SERPINA1 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, and wherein said second subunit binds to a second recognition half-site of said recognition sequence and comprises a second hypervariable (HVR2) region, wherein said engineered meganuclease comprises the amino acid sequence of any one of SEQ ID NOs:
- 17-22.
An engineered meganuclease that binds and cleaves a recognition sequence comprising SEQ ID NO:
- 11 within a SERPINA1 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, and wherein said second subunit binds to a second recognition half-site of said recognition sequence and comprises a second hypervariable (HVR2) region, wherein said engineered meganuclease comprises the amino acid sequence of any one of SEQ ID NOs:
- 41-46.
An engineered meganuclease that binds and cleaves a recognition sequence comprising SEQ ID NO:
- 13 within a SERPINA1 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, and wherein said second subunit binds to a second recognition half-site of said recognition sequence and comprises a second hypervariable (HVR2) region, wherein said engineered meganuclease comprises the amino acid sequence of any one of SEQ ID NOs:
- 65-71.
An engineered meganuclease that binds and cleaves a recognition sequence comprising SEQ ID NO:
- 15 within a SERPINA1 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, and wherein said second subunit binds to a second recognition half-site of said recognition sequence and comprises a second hypervariable (HVR2) region, wherein said engineered meganuclease comprises the amino acid sequence of any one of SEQ ID NOs:
- 93-100.
Dependencies are inferred from the claim text.