	US 12,440,579 B2
	Optimised RAG1 deficient gene therapy
Frank J. T. Staal, Leiden (NL); Karin Pike-Overzet, Leiden (NL); Laura Garcia-Perez, Leiden (NL); Marja C. J. A. Van Eggermond, Leiden (NL); and Arjan C. Lankester, Leiden (NL)
Assigned to ACADEMISCH ZIEKENHUIS LEIDEN (H.O.D.N. LUMC), Leiden (NL)
Appl. No. 17/438,135
Filed by ACADEMISCH ZIEKENHUIS LEIDEN (H.O.D.N. LUMC), Leiden (NL)
PCT Filed Mar. 6, 2020, PCT No. PCT/NL2020/050152 § 371(c)(1), (2) Date Sep. 10, 2021, PCT Pub. No. WO2020/185073, PCT Pub. Date Sep. 17, 2020.
Claims priority of application No. 2022714 (NL), filed on Mar. 11, 2019.
Prior Publication US 2022/0160894 A1, May 26, 2022
Int. Cl. A61K 48/00 (2006.01); C12N 15/86 (2006.01)

CPC A61K 48/005 (2013.01) [C12N 15/86 (2013.01); C12N 2740/16043 (2013.01); C12N 2800/22 (2013.01)]

20 Claims

1. A self-inactivating (SIN) lentiviral plasmid comprising an expression cassette comprising a promoter operably linked to a RAG1 transgene that comprises the nucleic acid sequence set forth by SEQ ID NO: 4, wherein the promoter is the MND promoter.