Abstract: Provided herein are MAPT RNAi agents and compositions comprising a MAPT RNAi agent. Also provided herein are methods of using the MAPT RNAi agents or compositions comprising a MAPT RNAi agent for reducing MAPT expression and/or treating tauopathy in a subject.

Abstract: The present invention provides for a double stranded RNA comprising a first RNA sequence and a second RNA sequence wherein the first and second RNA sequence are substantially complementary, wherein the first RNA sequence has a sequence length of at least 19 nucleotides and is substantially complementary to SEQ ID NO. 1. Said double stranded RNA is for use in inducing RNAi against Huntingtin exon 1 sequences. The double stranded RNA of to the invention was capable of reducing neuronal cell death and huntingtin aggregates in an animal model.

Abstract: Engineered dendritic cell vaccines, and methods of forming and applying same, that may be used as effective immunotherapies for cancers.

Abstract: This invention provides a nucleic acid that enables evaluation of various endocrine disrupting actions of a very small amount of endocrine disrupting chemicals with high sensitivity. Such nucleic acid comprises a total of 20 to 60 nucleotides comprising the nucleotide sequence shown in SEQ ID NO: 1 and shows excellent responsiveness to various endocrine disrupting chemicals.

Abstract: Compositions and methods useful to reduce expression of methylation-controlled J-protein (MCJ) gene and for treatment of MCJ-associated diseases and conditions are provided. Provided are MCJ dsRNA agents, MCJ antisense polynucleotide agents, compositions comprising MCJ dsRNA agents, and, compositions comprising MCJ antisense polynucleotide agents that can be used to reduce MCJ expression in cells and subjects.

Abstract: The present disclosure provides methods of identifying non-productive splice sites in target RNA transcripts and antisense oligonucleotides that increase the expression of said target RNA transcripts. In an embodiment, the target RNA transcript comprises ADAR, ARSA, ATPIA2, CACNAIA, DNMI, EIF2BI, EIF2B2, EIF2B5, IDUA, MFSD8, NF2, NPC1L PEXI, PRICKLE2, PRRT2, RAM, SETD5, SHANKS, SLC6A1, STXBPI, STX1B, and TCF4.

Abstract: The present disclosure relates to treatment of bleeding disorders, in particular, hemophilia A (mild, moderate or severe hemophilia) and von Willebrand disease (VWD), Type 1 or Type 2 or Type 3, using VWF targeting agents such as a PEGylated anti-VWF aptamer, BT200.

Abstract: The present invention includes methods for treating a disease or disorder that is associated with aberrant or absent dystrophin, by upregulating a brain isoform of dystrophin (e.g. purkinje and/or cortical) in a subject in need thereof.

Abstract: The present invention provides a composition and a method for efficiently delivering a nucleic acid agent to the central nervous system and/or the retina and bringing about antisense effects. Provided is a composition for reducing the expression level of a target transcription product in the central nervous system and/or retina of a subject, the composition including a nucleic acid complex that includes a first nucleic acid strand and a second nucleic acid strand, wherein: the first nucleic acid strand comprises a base sequence capable of hybridizing with at least part of the target transcription product and has an antisense effect on the target transcription product; the second nucleic acid strand comprises a base sequence complementary to the first nucleic acid strand and is conjugated to tocopherol, cholesterol, or an analog thereof; and the first nucleic acid strand is annealed to the second nucleic acid strand.

Abstract: Embodiments of the present disclosure concerns methods and compositions related to treatment using a CRISPR/Cas9/targeting antibody/gene targeting small guide RNA (sgRNA) fusion protein/ribonucleotide complex. The targeting of a polynucleotide of interest, such as a gene, may be effective to eradicate tumors in mammals. In some cases, sgRNA ribonucleotides with sequences designed to target any gene or genomic region are contemplated. In some embodiments, the targeting of SRC-3 in T regulatory cells in particular is effective to eradicate tumors in mammals. In specific cases, the T regulatory cells are subjected to CRISPR-based gene modification in vivo obviating the need for adoptive cell therapy. In some cases, substitutions of the CD25 targeting antibody is replaced with another antibody to recognize other cell surface antigens. In some cases, sgRNA ribonucleotides with sequences designed to target any gene or genomic region are described.

Abstract: Glaucoma or pathogenic intraocular pressure is treated by locally administering to an eye in need thereof formulations of a Wnt5a inhibitor.

Abstract: Among other things, the present disclosure provides C9orf72 oligonucleotides, compositions, and methods thereof. In some embodiments, the present disclosure provides methods for treating C9orf72-associated conditions, disorders or diseases, such as amyotrophic lateral sclerosis and frontotemporal dementia.

Abstract: A composition of matter includes an antisense phosphorodiamidate morpholino oligomer (MO) that includes an MO base sequence. The MO base sequence includes at least the base sequence gcagcccctggaacat (SEQ ID NO: 8) and is arranged to bind a corresponding complementary base sequence of messenger RNA (mRNA) transcribed from one or more genes for the beta subunit of human chorionic gonadotropin (hCG-?). An inventive method, for suppressing intracellular synthesis of a beta subunit of human chorionic gonadotropin (hCG-?), includes introducing such an MO into one or more cells.

Abstract: The embodiments of the present disclosure relate to one or more compositions or methods that upregulate the production of one or more sequences of micro-interfering ribonucleic acid (miRNA). The sequences of miRNA may be complimentary to a sequence of target messenger RNA (mRNA) that encodes for the translation of a target biomolecule, such as cyclic GMP-AMP synthase (cGAS). The miRNA can cause the target mRNA to be degraded or inactivated, thereby causing a decrease in bioavailability of the target biomolecule because it is degraded or inactivated by the miRNA. Decreasing the bioavailability of the target biomolecule within a subject that is administered the one or more compositions may address the afflictions experienced by the subject due to expression of the target biomolecule.

Abstract: The present invention relates to a RNA expression modulating or editing method through chemical or optogenetic regulation of Cas13 protein activity. Specifically, in order to regulate the activity of Cas13 in the CRISPR-Cas13 system, a fragment of the Cas13 protein was generated to enable recombination, and a chemogenetically or optogenetically bindable protein was linked to each fragment. And, it was confirmed that the Cas13 protein can be activated by treatment of a low molecular weight compound or irradiation of light. Accordingly, the method of the present invention can be effectively used for disease treatment by regulating RNA expression or editing RNA mutations related to disease.

Abstract: The present invention provides, among other things, methods of treating cystic fibrosis, comprising a step of administering to a subject in need of treatment a composition comprising an mRNA encoding a Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein, wherein the mRNA encoding the CFTR protein comprises a polynucleotide sequence at least 80% identical to SEQ ID NO: 1, wherein the mRNA is at a concentration of at least 0.4 mg/mL, and wherein the step of administering comprises inhalation.

Abstract: This invention relates to nanofibers comprising aptamers, which have increased stability and activity relative to free aptamers. The invention further relates to kill-switch nanofibers which disrupt the aptamer nanofibers. The invention further relates to methods of using the aptamer nanofibers and the kill-switch nanofibers to regulate the activity of extracellular targets recognized by the aptamers.

Abstract: This disclosure relates to DNA aptamers that bind eosinophil peroxidase, including uses thereof, such as in eosinophil peroxidase detection assays. Also provided is a method for detecting the presence of eosinophil peroxidase in a sample, using the DNA aptamers that bind eosinophil peroxidase. Further provided is a kit for detecting the presence of eosinophil peroxidase in a sample, using the DNA aptamers that bind eosinophil peroxidase.

Abstract: The present disclosure features useful compositions and methods to treat disorders for which deamination of an adenosine in an RNA produces a therapeutic result in a subject in need thereof.

Abstract: Provided are a glypican-3 (GPC3)-specific modified aptamer, and prevention or treatment of hepatoma using the same. The glypican-3-specific modified aptamer of the present invention specifically binds to glypican-3 to be internalized into hepatoma cells, and exhibits anticancer activity through an anticancer agent bound to the aptamer, and therefore, has a selective anticancer effect only for GPC3-expressing hepatoma cells without affecting normal hepatocytes.