Viral vector combining gene therapy and genome editing approaches for gene therapy of genetic disorders
AssigneesInstitut National De La Sante Et De La Recherche Medicale (Inserm), Universite Paris Descartes, Assistance Publique-Hopitaux de Paris, Magine - Institut Des Maladies Genetiques Necker Enfants Malades
InventorsAnnarita MICCIO, Vasco MENEGHINI
Life of this patent
Estimating the term…
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 Cpf1 protein, methods for modifying the genome of a cell, and the cells obtainable by such methods.
Claims41
A recombinant viral vector comprising in its genome:
- (i) a nucleotide sequence encoding a guide RNA (gRNA) that comprises a spacer adapted to bind to a target nucleotide sequence, said target nucleotide sequence is within the coding sequence of a target gene, within a transcribed non-coding sequence of a target gene or within a non-transcribed sequence, either upstream or downstream, of a target gene, said target gene is involved in a genetic disorder;
- and
- (ii) a nucleotide sequence encoding a protein that has a therapeutic effect in said genetic disorder.
Dependencies are inferred from the claim text.