Treatment of sos2 related diseases and disorders
AssigneeEmpirico Inc.
InventorsOmri GOTTESMAN, Shannon BRUSE, Brian CAJES, David JAKUBOSKY, Sarah E. KLEINSTEIN, John VEKICH
Life of this patent
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Abstract
Disclosed herein are compositions comprising an oligonucleotide that targets SOS Ras/Rho guanine nucleotide exchange factor 2 (SOS2). The oligonucleotide may include a small interfering RNA (siRNA) or an antisense oligonucleotide (ASO). Also provided herein are methods of treating conditions associated with SOS2 mutations that include providing an oligonucleotide that targets SOS2 to a subject.
Claims25
A composition comprising an oligonucleotide that targets SOS2 and when administered to a subject in an effective amount increases an estimated glomerular filtration rate, or decreases a creatinine, blood urea nitrogen, proteinuria, microalbuminuria, blood urate, systolic or diastolic blood pressure, intraocular pressure, hemoglobin A1C, alanine aminotransferase, aspartate aminotransferase, or liver fat percentage measurement.
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A composition comprising an oligonucleotide that inhibits the expression of SOS2 wherein the oligonucleotide comprises an siRNA comprising a sense strand and an antisense strand, each strand is independently about 12-30 nucleosides in length, and at least one of the sense strand and the antisense strand comprises a nucleoside sequence comprising about 12-30 contiguous nucleosides of SEQ ID NO:
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26-27. (canceled)
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Dependencies are inferred from the claim text.