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1. (WO2018220210) RECOMBINANT LENTIVIRAL VECTOR FOR STEM CELL-BASED GENE THERAPY OF SICKLE CELL DISORDER

Pub. No.:    WO/2018/220210    International Application No.:    PCT/EP2018/064531
Publication Date: Fri Dec 07 00:59:59 CET 2018 International Filing Date: Sat Jun 02 01:59:59 CEST 2018
IPC: C12N 15/86
C12N 15/87
C07K 14/805
Applicants: INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM)
UNIVERSITE PARIS DESCARTES
ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS
IMAGINE - INSTITUT DES MALADIES GENETIQUES NECKER ENFANTS MALADES
Inventors: MICCIO, Annarita
MENEGHINI, Vasco
Title: RECOMBINANT LENTIVIRAL VECTOR FOR STEM CELL-BASED GENE THERAPY OF SICKLE CELL DISORDER
Abstract:
This invention relates to recombinant lentiviral vectors, compositions thereof, the use of the vectors or the compositions thereof, kits of parts comprising said vectors or compositions thereof and a catalytically active Cas9 or Cpf1 protein, methods for modifying the genome of a hematopoietic stem/progenitor cell (HSPC), and the HSPC obtainable by such methods.