美國
US 12,697,400 B2
Gene editing methods for treating alpha-1 antitrypsin (AAT) deficiency
US 12,697,400 B2 是美國專利商標局（USPTO）的核准專利，日期 2026-08-04。申請人：Precision BioSciences, Inc.。PDF 可在本頁下載，未登入每天最多 5 篇。
- 申請人
- Precision BioSciences, Inc.
- 發明人
- James Jefferson Smith; John Morris; Janel Lape; Cassandra Gorsuch; Paige Scarlett Nemec; Jason Richard Harris; Wendy Shoop
- 公開日
- 2026-08-04
- 申請日
- 2024-04-18
- 文件種類
- B2 核准公告
- 也寫作
- US 12,697,400 B2 · US12697400 · 12697400
摘要
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.
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專利局網站： 美國專利商標局（USPTO）