Methods and compositions for delivery of biologics
AssigneeSangamo Therapeutics, Inc.
InventorGregory J. Cost
Life of this patent
Estimating the term…
Abstract
Nucleases and methods of using these nucleases for genetic alteration of red blood cells (RBCs), for example for providing for a protein lacking in a monogenic disorder or a biologic for the treatment of exposure to a toxin using genetically altered RBCs.
Claims6
An isolated modified type O negative (type O−) red blood cell (RBC) that secretes a protein that degrades or detoxifies one or more nerve agents, wherein the RBC is derived from a blood type O-negative population of RBC precursor cells comprising (i) a pair of zinc finger nucleases (ZFNs) that cleave an endogenous beta globin gene and (ii) a transgene comprising a sequence encoding the protein, wherein the transgene is integrated into the endogenous beta globin gene of the RBC precursor cell population following cleavage by the pair of ZFNs, and further wherein the transgene integrated into the endogenous beta globin gene is integrated into or between the target sites as shown in SEQ ID NO:1 and SEQ ID NO:2 or into or between the target sites as shown in SEQ ID NO:13 and SEQ ID NO:14, such that expression of the protein is driven by the endogenous promoter of the beta globin gene.
Dependencies are inferred from the claim text.