US 12,234,490 B2
Composition for prime editing comprising pegRNA for editing mutation of alpha-1 antitrypsin deficiency
Hyongbum Henry Kim, Seoul (KR); Minyoung Lee, Seoul (KR); and Yoo Jin Chang, Seoul (KR)
Assigned to INDUSTRY-ACADEMIC COOPERATION FOUNDATION, YONSEI UNIVERSITY, Seoul (KR)
Filed by INDUSTRY-ACADEMIC COOPERATION FOUNDATION, YONSEI UNIVERSITY, Seoul (KR)
Filed on Feb. 16, 2023, as Appl. No. 18/170,035.
Claims priority of application No. 10-2022-0021737 (KR), filed on Feb. 18, 2022.
Prior Publication US 2023/0332121 A1, Oct. 19, 2023
Int. Cl. C12N 9/22 (2006.01); A61P 35/00 (2006.01); C12N 15/10 (2006.01); C12N 15/86 (2006.01)
CPC C12N 9/22 (2013.01) [A61P 35/00 (2018.01); C12N 15/102 (2013.01); C12N 15/86 (2013.01); C12N 2310/20 (2017.05); C12N 2740/15043 (2013.01)] 5 Claims
OG exemplary drawing
 
1. A composition for editing a Z mutation of an SERPINA1 gene, comprising: a recombinant vector expressing a prime editor protein, the prime editor protein comprising a nucleic acid sequence encoding a Cas9 nickase-reverse transcriptase; and a recombinant vector comprising a prime editing guide RNA (pegRNA) sequence, wherein each respective recombinant vector is selected from the group consisting of a lentiviral vector, a retroviral vector, an adenoviral vector, and a herpes simplex viral vector, wherein the nucleic acid sequence encoding the Cas9 nickase-reverse transcriptase consists of the nucleotide sequence of SEQ ID NO: 6, and wherein the pegRNA sequence consists of the nucleotide sequence of SEQ ID NO: 1.