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AssigneeBoard of Regents, The University of Texas System
InventorsRonak LAKHIA, Harini RAMALINGAM, Laurence BIGGERS, Maggie ZUMWALT, Patricia COBO-STARK, Vishal D. PATEL
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The present disclosure provides in part an antisense oligonucleotide strategy to increase levels of polycystin 1 and/or polycystin 2 (PKD1 and/or PKD2). By targeting and inhibiting regulatory elements of the 3′ UTR of PKD1 mRNA and/or PKD2 mRNA, compositions of the present disclosure are able to de-repress PKD1 and/or PKD2 mRNA and increase expression of PKD1 and/or PKD2 protein. Further methods are provided for increasing PKD1 and/or PKD2 expression in cells in vitro or in vivo as well as methods of treating autosomal dominant polycystic kidney disease using the antisense oligonucleotides provided herein.
A method of selectively increasing expression of polycystin 1 and/or polycystin 2 in a cell, the method comprising delivering an antisense oligonucleotide (ASO) that hybridizes to a 3′UTR regulatory region on an mRNA encoding polycystin 1 or polycystin 2 (PKD1 or PKD2 mRNA to the cell.
A method of treating Autosomal Dominant Polycystic Kidney Disease (ADPKD) in a subject in need thereof, the method comprising administering a pharmaceutically effective amount of an antisense oligonucleotide (ASO) that hybridizes to a 3′UTR regulatory region on an mRNA encoding polycystin 1 or polycystin 2 (PKD1 or PKD2 mRNA to the subject.
An antisense oligonucleotide (ASO) that hybridizes to a 3′UTR regulatory region on an mRNA encoding polycystin 1 or polycystin 2 (PKD1 or PKD2 mRNA).
Dependencies are inferred from the claim text.