US 12,466,864 B2
Production of large-sized quasidystrophins using overlapping AAV vectors
Isabelle Richard, Corbeil Essonnes (FR); and William Lostal, Savigny sur Orge (FR)
Assigned to GENETHON, Evry-Courcouronnes (FR); INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE), Paris (FR); and UNIVERSITE D'EVRY-VAL-D'ESSONNE, Evry-Courcouronnes (FR)
Appl. No. 17/593,285
Filed by GENETHON, Evry-Courcouronnes (FR); INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE), Paris (FR); and UNIVERSITE D'EVRY-VAL-D'ESSONNE, Evry-Courcouronnes (FR)
PCT Filed Mar. 25, 2020, PCT No. PCT/EP2020/058372
§ 371(c)(1), (2) Date Sep. 14, 2021,
PCT Pub. No. WO2020/193636, PCT Pub. Date Oct. 1, 2020.
Claims priority of application No. 19305377 (EP), filed on Mar. 25, 2019.
Prior Publication US 2022/0204574 A1, Jun. 30, 2022
Int. Cl. C12N 15/86 (2006.01); A61K 35/76 (2015.01); A61P 21/00 (2006.01); C07K 14/47 (2006.01); C12N 5/077 (2010.01); A61K 38/00 (2006.01)
CPC C07K 14/4708 (2013.01) [A61K 35/76 (2013.01); A61P 21/00 (2018.01); C12N 5/0652 (2013.01); C12N 15/86 (2013.01); A61K 38/00 (2013.01); C12N 2750/14143 (2013.01); C12N 2750/14145 (2013.01); C12N 2750/14171 (2013.01)] 10 Claims
OG exemplary drawing
 
1. A dual AAV vector system comprising two AAV vectors, wherein
a first AAV vector comprises, between 5′ and 3′ AAV ITRs, a first nucleic acid sequence that encodes a N-terminal part of a quasidystrophin, and
a second AAV vector comprises, between 5′ and 3′ AAV ITRs, a second nucleic acid sequence that encodes a C-terminal part of a quasidystrophin,
wherein the first and second nucleic acid sequences comprise an overlapping region that permits the production by recombination of the quasidystrophin, wherein the quasidystrophin is encoded by a sequence according to SEQ ID NO: 22.