	US 12,012,613 B2
	Genetically engineered hematopoietic stem cell as a platform for systemic protein expression
Mario Amendola, Paris (FR); and Giulia Pavani, Paris (FR)
Assigned to Genethon, Paris (FR); and Institut National De La Sante Et De La Recherche Medicale, Paris (FR)
Appl. No. 16/961,483
Filed by GENETHON, Evry (FR); UNIVERSITE D'EVRY-VAL-D'ESSONE, Evry (FR); and INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM), Paris (FR)
PCT Filed Jan. 11, 2019, PCT No. PCT/EP2019/050710 § 371(c)(1), (2) Date Jul. 10, 2020, PCT Pub. No. WO2019/138082, PCT Pub. Date Jul. 18, 2019.
Claims priority of application No. 18305026 (EP), filed on Jan. 12, 2018.
Prior Publication US 2020/0377857 A1, Dec. 3, 2020
Int. Cl. C12N 5/0789 (2010.01); A61K 35/28 (2015.01); C12N 9/22 (2006.01); C12N 15/113 (2010.01)

CPC C12N 5/0647 (2013.01) [A61K 35/28 (2013.01); C12N 9/22 (2013.01); C12N 15/113 (2013.01); C12N 2310/20 (2017.05); C12N 2800/80 (2013.01)]

13 Claims

1. A genetically modified hematopoietic stem cell comprising, in at least one globin gene comprised in the genome thereof, at least one transgene encoding a therapeutic protein or a therapeutic ribonucleic acid, the transgene being placed under control of the endogenous promoter of the at least one globin gene,

wherein the at least one transgene encoding a therapeutic protein or a therapeutic ribonucleic acid is comprised in the 5′ region, in the 3′ untranslated region (3′ UTR) and/or in an intron of the at least one globin gene,

wherein the at least one globin gene is selected from the group consisting of the alpha 1 globin gene and the alpha 2 globin gene.