The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.
The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.
Patent No.:
Date of Patent:
Feb. 25, 2025
Filed:
Feb. 16, 2023
Industry-academic Cooperation Foundation, Yonsei University, Seoul, KR;
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.