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US 2025/0144183 A1Published application
Methods and compositions for treating lipoprotein-related diseases
OwnerSanjay D'SOUZA, Laura SERWER +2inventors
InventorsSanjay D'SOUZA, Laura SERWER, Jonathan TERRETT, John KULMAN
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Abstract
The present disclosure relates to methods, compositions and kits for modulating the expression of LPA gene and for treating lipoprotein-related diseases, for example cardiovascular diseases, in a subject by gene editing.
Claims21
1-53. (canceled)
A method for treating a lipoprotein-related disease in a subject in need thereof, comprising administering to the subject a plurality of lipid nanoparticles comprising (a) a guide RNA (gRNA) targeting LPA gene (LPA gRNA) or a nucleic acid encoding the LPA gRNA, wherein the LPA gRNA comprises a spacer sequence of SEQ ID NO:
- 18, and (b) a nucleic acid encoding SpCas9;
- wherein (a) and (b) are encapsulated in the plurality of lipid nanoparticles;
- wherein the method comprises one or two administrations of the plurality of lipid nanoparticles, and the reduction of concentration of Lp(a) protein in the plasma of the subject is at least 65% sixty days after the administrations of the plurality of lipid nanoparticles;
- and thereby treating the lipoprotein-related disease in the subject.
1 more element
Dependencies are inferred from the claim text.