Liposomes for modulating Wiskott-Aldrich syndrome protein
AssigneeBar-Ilan University
InventorMira Barda-Saad
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Abstract
Embodiments of the invention relate to liposomes comprising: a lipid bilayer having an internal cavity; a therapeutic agent within the internal cavity configured to modify expression or degradation of WASp in a cell; and a targeting moiety external to the lipid bilayer configured to target an extracellular domain of a cell. Embodiments of the invention relate to methods of treatment of disease comprising administering the liposomes. Novel pharmaceutical compositions are also disclosed.
Claims12
A method for the treatment of a disease in a patient suffering from a condition selected from the group consisting of a hematopoietic malignancy, Wiskott-Aldrich syndrome (WAS), and X-linked thrombocytopenia (XLT) using a therapeutic agent that modifies expression or degradation of WASp in a cell, the method comprising:
- administering to said patient a liposome or a pharmaceutical composition comprising said liposome, wherein said liposome comprises a lipid bilayer having an internal cavity, a therapeutic agent within the internal cavity configured to modify expression or degradation of WASp in a cell, and a targeting moiety external to the lipid bilayer configured to target an extracellular domain of a cell and wherein said therapeutic agent is (a) a WASp specific-siRNA that, when contacted with a cell, reduces the expression of WASp or increases WASp degradation in said cell;
- or (b) a peptide that binds to WASp at lysine residues 76 or 81, thereby protecting WASp from ubiquitylation and subsequent degradation.
Dependencies are inferred from the claim text.