United States
US 12,697,400 B2
Gene editing methods for treating alpha-1 antitrypsin (AAT) deficiency
US 12,697,400 B2 is a granted patent from the United States Patent and Trademark Office (USPTO), dated 2026-08-04. Applicant: Precision BioSciences, Inc. Its PDF downloads from this page without an account, up to 5 a day.
- Applicant
- Precision BioSciences, Inc.
- Inventors
- James Jefferson Smith; John Morris; Janel Lape; Cassandra Gorsuch; Paige Scarlett Nemec; Jason Richard Harris; Wendy Shoop
- Publication date
- 2026-08-04
- Filing date
- 2024-04-18
- Document type
- B2 Granted patent
- Also written as
- US 12,697,400 B2 · US12697400 · 12697400
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.
The opening of the abstract. The full text is in the PDF.
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Patent office website: United States Patent and Trademark Office (USPTO)
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