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1. (WO2018220211) VIRAL VECTOR COMBINING GENE THERAPY AND GENOME EDITING APPROACHES FOR GENE THERAPY OF GENETIC DISORDERS

Pub. No.:    WO/2018/220211    International Application No.:    PCT/EP2018/064532
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
A61K 35/76
C12N 15/87
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: VIRAL VECTOR COMBINING GENE THERAPY AND GENOME EDITING APPROACHES FOR GENE THERAPY OF GENETIC DISORDERS
Abstract:
This invention relates to recombinant viral vectors, preferably retroviral (RV), lentiviral (LV) or adeno-associated viral (AAV) vectors, compositions thereof, the use of the recombinant viral vectors or the compositions thereof, kits of parts comprising said recombinant viral vectors or compositions thereof and a catalytically active Cas9 or Cpfl protein, methods for modifying the genome of a cell, and the cells obtainable by such methods.