Provided herein are methods for the treatment of polycystic kidney disease, including autosomal dominant polycystic kidney disease, using modified oligonucleotides targeted to miR-17.

Provided is a polymerizable compound represented by a Formula E-1 or Formula E-2 shown in the description. In Formula E-1 or Formula E-2, R.sup.1 represents an alkoxy group, —NR.sup.N.sub.2, a hydroxy group, an aryl group, or an alkyl group, wherein R.sup.N each independently represents a hydrogen atom or an alkyl group having 1 to 10 carbon atoms; n represents an integer from 1 to 5; R.sup.3 represents a hydrogen atom, an acetyl group, a phenoxyacetyl group, a pivaloyl group, a benzyl group, a 4-methoxybenzyl group, a benzoyl group, a triphenylmethyl group, a 4,4′-dimethoxytrityl (DMTr) group, a 4-methoxytrityl (MMTr) group, a 9-phenylxanthenyl group, a trimethylsilyl group, a cyanomethoxymethyl group, a 2-(cyanoethoxy)ethyl group, or a cyanoethoxymethyl group; and X represents a structure represented by any one of Formula B-1 to Formula B-5 shown in the description.

Provided is a polymerizable compound represented by a Formula E-1 or Formula E-2 shown in the description. In Formula E-1 or Formula E-2, R.sup.1 represents an alkoxy group, —NR.sup.N.sub.2, a hydroxy group, an aryl group, or an alkyl group, wherein R.sup.N each independently represents a hydrogen atom or an alkyl group having 1 to 10 carbon atoms; n represents an integer from 1 to 5; R.sup.3 represents a hydrogen atom, an acetyl group, a phenoxyacetyl group, a pivaloyl group, a benzyl group, a 4-methoxybenzyl group, a benzoyl group, a triphenylmethyl group, a 4,4′-dimethoxytrityl (DMTr) group, a 4-methoxytrityl (MMTr) group, a 9-phenylxanthenyl group, a trimethylsilyl group, a cyanomethoxymethyl group, a 2-(cyanoethoxy)ethyl group, or a cyanoethoxymethyl group; and X represents a structure represented by any one of Formula B-1 to Formula B-5 shown in the description.

The present disclosure features useful compositions and methods to treat disorders for which deamination of an adenosine in an mRNA produces a therapeutic result,

Disclosed herein are compounds including a single-stranded oligonucleotide (A) having a nucleobase sequence complementary to a portion of the dystrophin pre-mRNA, their preparation, and uses thereof for the treatment of Duchenne muscular dystrophy.

The present invention relates to a polynucleotide comprising a Nuclear factor of activated T-cells (NFAT) binding site sequence and a reverse complement of said NFAT binding site sequence separated by a spacer sequence, to said polynucleotide for use in treating and/or preventing disease, and to viral particles, compositions, and uses related thereto. The present invention further relates to a polynucleotide comprising a Nuclear factor of activated T-cells (NFAT) binding site sequence and a reverse complement of said NFAT binding site sequence for use in treating and/or preventing an NFAT-mediated disease.

The present invention relates to a polynucleotide comprising a Nuclear factor of activated T-cells (NFAT) binding site sequence and a reverse complement of said NFAT binding site sequence separated by a spacer sequence, to said polynucleotide for use in treating and/or preventing disease, and to viral particles, compositions, and uses related thereto. The present invention further relates to a polynucleotide comprising a Nuclear factor of activated T-cells (NFAT) binding site sequence and a reverse complement of said NFAT binding site sequence for use in treating and/or preventing an NFAT-mediated disease.

The present disclosure features useful compositions and methods to treat disorders for which deamination of an adenosine in an mRNA produces a therapeutic result, e.g., in a subject in need thereof.

The present disclosure features useful compositions and methods to treat disorders for which deamination of an adenosine in an mRNA produces a therapeutic result, e.g., in a subject in need thereof.

There is provided a method of synthesizing a single-stranded nucleotide sequence, the method comprising adding a blocked nucleoside triphosphate to an initiator nucleotide sequence to incorporate a corresponding blocked nucleotide thereto in the presence of a polymerase, wherein the blocked nucleoside triphosphate has one of the general formulae (I), (II), (III), (IV), (V) and (VI).