{"number":"US12697400B2","kindCode":"B2","documentType":"grant","title":{"original":"Gene editing methods for treating alpha-1 antitrypsin (AAT) deficiency","english":"Gene editing methods for treating alpha-1 antitrypsin (AAT) deficiency"},"abstract":{"original":"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.","english":"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."},"applicants":["Precision BioSciences, Inc."],"inventors":["James Jefferson Smith","John Morris","Janel Lape","Cassandra Gorsuch","Paige Scarlett Nemec","Jason Richard Harris","Wendy Shoop"],"filingDate":"2024-04-18","publicationDate":"2026-08-04","grantDate":"2026-08-04","verifiedWithOffice":true,"pdfUrl":"https://downloadpatent.com/pdf/US12697400B2.pdf","pages":{"en":"https://downloadpatent.com/patent/US12697400B2","zh-Hant":"https://downloadpatent.com/zh-Hant/patent/US12697400B2","ja":"https://downloadpatent.com/ja/patent/US12697400B2"}}