Lpl variant therapeutics
AssigneesMichael R. Hayden, John P. Kastelein, Katherine Excoffon
InventorsMichael R. HAYDEN, John P. Kastelein
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Abstract
The invention provides for the use of a therapeutic derived from a truncated lipoprotein lipase protein (LPL S447X), including nucleic acids encoding such proteins, for the treatment of conditions including LPL responsive conditions, such as cardiovascular disease, hypertension, LPL deficiency, high triglyceride levels, low HDL-cholesterol levels or atherosclerosis.
Claims34
The use of an LPL S447X therapeutic for the preparation of a pharmaceutical composition for the treatment of an LPL-responsive condition in a subject.
A method of treating a disease in a subject, comprising administering to the subject an effective amount of an LPL S447X therapeutic, wherein the disease is an LPL-responsive disease.
An LPL S447X therapeutic for use as an active pharmaceutical substance, wherein the LPL S447X therapeutic is selected from the group consisting of:
- a) an LPL S447X protein, wherein the amino acid sequence of the LPL S447X protein comprises a contiguous segment having at least 90% sequence identity to SEQ ID NO:3 when optimally aligned, and wherein the LPL S447X protein lacks amino acids corresponding to amino acids 447 and 448 of SEQ ID NO:3 when optimally aligned;
- and,
- b) an LPL S447X nucleic acid encoding the LPL S447X protein.
A gene therapy vector comprising an LPL S447X therapeutic, wherein the LPL S447X therapeutic is selected from the group consisting of:
- a) an LPL S447X protein, wherein the amino acid sequence of the LPL S447X protein comprises a contiguous segment having at least 90% sequence identity to SEQ ID NO:3 when optimally aligned, and wherein the LPL S447X protein lacks amino acids corresponding to amino acids 447 and 448 of SEQ ID NO:3 when optimally aligned;
- and,
- b) an LPL S447X nucleic acid encoding the LPL S447X protein.
An LPL S447X therapeutic substantially as hereinbefore described and with reference to the examples and drawings.
Dependencies are inferred from the claim text.