US 11,891,617 B1
Modified complex platform of adeno-associated virus with improved rate of expression of loaded genes and reduced genotoxicity
Suk Chul Bae, Cheongju-si (KR); You Soub Lee, Cheongju-si (KR); Xinzi Chi, Cheongju-si (KR); Tae Geun Park, Cheongju-si (KR); and Woo-Jin Kim, Seoul (KR)
Assigned to GENECRAFT INC., Cheongju-si (KR)
Filed by GENECRAFT INC., Cheongju-si (KR)
Filed on Mar. 27, 2023, as Appl. No. 18/190,630.
Int. Cl. C12N 7/01 (2006.01); C12N 15/86 (2006.01)
CPC C12N 15/86 (2013.01) [C12N 2750/14143 (2013.01); C12N 2750/14145 (2013.01)] 9 Claims
 
1. An adeno-associated virus (AAV) complex, comprising a polynucleotide sequence encoding a transgene between a first inverted terminal repeat (ITR) and a second ITR,
wherein the first ITR is not modified and the second ITR is modified,
wherein in the second ITR, all or part of a stem-loop structure, which is formed of rep-binding element (RBE), RBE′, A, A′, B, B′, C, C′, and D regions, is modified,
wherein the modified ITR exists as an open-end without forming any of the stem region and the loop region of the stem-loop structure.