Transient inhibition of p53 in gene therapy
AssigneesOspedale San Raffaele S.R.L., Fondazione Telethon Ets
InventorsPietro Genovese, Giulia Schiroli, Luigi Naldini, Aurelien Jacob, Samuele Ferrari
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Abstract
An agent which promotes homology directed DNA repair for use in haematopoietic stem and/or progenitor cell gene therapy, wherein said haematopoietic stem cells are gene edited. Use of an agent which promotes homology directed DNA repair, for increasing the survival and/or engraftment of gene edited haematopoietic stemand/or progenitorcells or for increasing the efficiency of gene editing of haematopoietic stem and/or progenitor cells.
Claims16
A method of haematopoietic cell gene therapy, haematopoietic stem cell gene therapy, and/or haematopoietic progenitor cell gene therapy, wherein said gene therapy comprises gene editing, wherein said method comprises contacting a population of haematopoietic cells, haematopoietic stem cells and/or haematopoietic progenitor cells with an agent that comprises a p53 dominant negative peptide comprising a p53 homo-oligomerisation domain, or that comprises a nucleic acid comprising a nucleotide sequence encoding the p53 dominant negative peptide, wherein the method transiently inhibits p53 in the haematopoietic cells, haematopoietic stem cells and/or haematopoietic progenitor cells.
Dependencies are inferred from the claim text.