Liposomes for modulating wiskott-aldrich syndrome protein
AssigneeBar-Ilan University
InventorMira BARDA-SAAD
Life of this patent
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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.
Claims20
A liposome 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, and wherein said therapeutic agent is selected from the group consisting of a peptide or an oligonucleotide.
A pharmaceutical composition comprising a pharmaceutically acceptable carrier and a therapeutic agent that modifies expression or degradation of WASp in a cell, 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.
A WASp specific-siRNA that, when contacted with a cell, reduces the expression of WASp or increases WASp degradation in said cell, wherein said siRNA comprises an oligonucleotide having the sequence of SEQ ID NO. 1, SEQ ID NO. 2, SEQ ID NO. 3, SEQ ID NO. 4, SEQ ID NO. 5, SEQ ID NO. 6 or SEQ ID NO. 7, and a duplex of said oligonucleotide with its complementary strand, or any mixture of said siRNAs.
A peptide that binds to WASp at lysine residues 76 or 81, thereby protecting WASp from ubiquitylation and subsequent degradation, the peptide comprising SEQ. ID NO. 13, 14 or 15.
Dependencies are inferred from the claim text.