Gene vector
AssigneesFondazione Telethon, Ospedale San Raffaele S.R.L.
InventorsAlessandra Biffi, Bernhard Rudolf Gentner, Luigi Naldini
Life of this patent
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Abstract
A gene vector for use in gene therapy comprising at least one miRNA sequence target operably linked to a nucleotide sequence having a corresponding miRNA in a hematopoietic progenitor cell (HSPC) or hematopoietic stem cell (HSC) which prevents or reduces expression of the nucleotide sequence in a HSPC or HSC but not in a differentiated cell.
Claims12
A hematopoietic stem or progenitor cell for use in gene therapy, wherein the cell is transduced or transfected with a gene vector comprising at least one mir-130a and/or mir-126 target sequence operably linked to a transgene, wherein the transgene encodes an immunomodulatory molecule, lysosomal enzyme galactocerebrosidase, or gp91 phox, and wherein expression of the transgene is prevented or reduced in the hematopoietic stem or progenitor cell but not in differentiated myeloid and lymphoid progeny of the hematopoietic stem or progenitor cell.
A hematopoietic stem or progenitor cell for use in gene therapy, wherein the cell is transduced or transfected with a gene vector comprising a TEK (Tie2) promoter and at least one mir-130a and/or mir-126 target sequence operably linked to a transgene encoding interferon-alpha, and wherein expression of the transgene is prevented or reduced in the hematopoietic stem or progenitor cell but not in differentiated myeloid and lymphoid progeny of the hematopoietic stem or progenitor cell.
Dependencies are inferred from the claim text.