Inhibition of nonsense mediated decay pathways
AssigneeUniversity of Miami
InventorEli Gilboa
Life of this patent
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Abstract
Compositions for inducing or enhancing antigenicity of a target cell by modulating the nonsense mediated decay pathway in the target cell. The compositions comprise one or more cell binding ligands providing specificity and delivery of an oligonucleotide or other molecule to the target. These compositions have broad applicability in the treatment of many diseases.
Claims27
A composition for inhibiting nonsense mediated decay (NMD) pathways in patients in vivo comprising at least one first domain which specifically binds to at least one tumor cell target or normal cell target in a tumor stroma and at least one second domain specific for a molecular component of nonsense mediated decay pathways, wherein the second domain comprises an antisense oligonucleotide molecule, peptides, proteins, nucleic acids, organic or inorganic molecules, which inhibit the nonsense mediated decay pathway.
A composition for inducing novel antigens in abnormal cells, comprising:
- a multi-domain molecule having at least one target specific domain and at least one domain, which modulates expression and function of molecules associated with nonsense mediated decay pathways.
A method of inducing or enhancing immunogenicity of a target cell in vitro or in vivo and modulating an antigen specific immune response in patient comprising:
- obtaining a composition comprising at least target binding molecule conjugated to at least one oligonucleotide molecule wherein the target binding ligand selectively binds to tumor cell or normal cell target ligands of tumor stroma and the oligonucleotide is specific for a molecule associated with at least one factor associated with a nonsense mediated decay pathway (NMD);
- administering the composition in a therapeutically effective amount to the patient;
- and,
- inducing or enhancing the immunogenicity of a target cell and modulating an antigen specific immune response in patient.
A method of up-regulating existing and/or inducing new or novel antigens on a cell's surface comprising contacting the cell with the composition comprising at least one first domain which binds to at least one tumor or normal cell target ligand in tumor stroma and at least one second domain specific for a molecular component of nonsense mediated decay pathways, wherein the NMD domain comprises an antisense oligonucleotide molecule, peptides, proteins, nucleic acids, organic or inorganic molecules which inhibit the nonsense mediated decay pathway.
A method of preventing or treating cancer in vivo, comprising administering to a patient a therapeutically effective amount of a composition comprising at least one first domain which binds to at least one tumor cell or normal cell target ligand in tumor stroma and at least one second domain specific for a molecular component of nonsense mediated decay pathways, wherein the NMD domain comprises an antisense oligonucleotide molecule, peptides, proteins, nucleic acids, organic or inorganic molecules which inhibit the nonsense mediated decay pathway.
Dependencies are inferred from the claim text.