公开(公告)号：US12234490B2

公开(公告)日：2025-02-25

申请号：US18170035

申请日：2023-02-16

Applicant: INDUSTRY-ACADEMIC COOPERATION FOUNDATION, YONSEI UNIVERSITY

Inventor： Hyongbum Henry Kim ,  Minyoung Lee ,  Yoo Jin Chang

IPC: C12N9/22 ,  A61P35/00 ,  C12N15/10 ,  C12N15/86

Abstract: Provided is a genome editing composition for prime editing including a prime editor protein and a prime editing guide RNA, for editing a Z-type mutation of alpha-1 antitrypsin deficiency. The composition according to an aspect includes both a pegRNA sequence capable of effectively editing the SERPINA1 gene and prime editor 2 (PE 2), and thus, may effectively deliver the prime editor to a cell, and act specifically for a target sequence of the SERPINA1 gene, enabling genome editing with high accuracy, and therefore, may be useful as a SERPINA1 gene editing platform. In addition, the composition is capable of correcting the Z-type mutation of the SERPINA1 gene, and may be used for treatment or prevention of alpha-1 antitrypsin deficiency.