Abstract: Disclosed herein are compounds, compositions and methods for modulating the expression of huntingtin in a cell, tissue or animal. Further provided are methods of slowing or preventing Huntington's disease progression using an antisense compound targeted to huntingtin. Additionally provided are methods of delaying or preventing the onset of Huntingtin's disease in an individual susceptible to Huntingtin's Disease. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders.

Abstract: The present invention relates to a portable analyzer for detecting bisphenol A, which comprises an aptamer specifically binding to bisphenol A, and a method for detecting bisphenol A using the same. The analyzer of the present invention can analyze a small amount of a sample collected from a contaminated environment in a field at a level similar to a laboratory environment, thereby having an effect of enabling a more immediate and accurate detection and quantification of bisphenol A.

Abstract: Among other things, the present disclosure relates to chirally controlled oligonucleotides of select designs, chirally controlled oligonucleotide compositions, and methods of making and using the same. In some embodiments, a provided chirally controlled oligonucleotide composition provides different cleavage patterns of a nucleic acid polymer than a reference oligonucleotide composition. In some embodiments, a provided chirally controlled oligonucleotide composition provides single site cleavage within a complementary sequence of a nucleic acid polymer. In some embodiments, a chirally controlled oligonucleotide composition has any sequence of bases, and/or pattern or base modifications, sugar modifications, backbone modifications and/or stereochemistry, or combination of these elements, described herein.

Abstract: Disclosed herein are compositions for linking DNA binding domains and cleavage domains (or cleavage half-domains) to form non-naturally occurring nucleases with alternative configurations. Also described are methods of making and using compositions comprising these linkers.

Abstract: Endoglin has been identified to play a functional role as a regulator of TGF?1 signaling, particular in TGF?1-mediated calcineurin expression. The present invention features methods of reducing cardiac damage, particularly in a subject undergoing chemotherapy or radiation therapy by administering a composition that inhibits endoglin activity. The present invention also features methods of treating autoimmune diseases, inflammatory diseases, organ transplantation, and conditions association with oxidative stress related to TGF?1-mediated calcineurin expression and reactive oxygen species (ROS) production by administering a composition that inhibits endoglin activity. The present invention also features methods of treating fibrotic diseases by administering a composition that inhibits endoglin activity.

Abstract: A pharmaceutical composition for preventing and treating metabolic diseases, which includes an MKRN1 expression or activity inhibitor as an active ingredient, is provided. Because the MKRN1 of the present invention functions as an E3 ligase, which ubiquitinates AMPK?, to degrade an AMPK protein, expression and activity levels of AMPK can be restored by suppressing the MKRN1 expression. Also, the MKRN1 expression or activity inhibitor of the present invention can be effectively used as an active ingredient of the composition for preventing and treating metabolic diseases because an improving effect on obesity, diabetes, and fatty liver can be exhibited by MKRN1 expression knockout in a mouse model in which the MKRN1 expression is knocked out and the obesity, diabetes and fatty liver are induced by high-fat diets.

Abstract: Aspects of the disclosure relate to methods of altering RNA splicing in a subject. In some embodiments, methods are provided for correcting splicing in a cell that contains a DYSF gene having a mutation that results in defective splicing.

Abstract: Disclosed are molecules for treating non-del(5q) MDS that mimic allelic deficiency in del5q MDS to sensitize the malignant clones of patient without del(5q). The disclosed molecule contains an inhibitor of Cdc25C, an inhibitor of PP2Ac?, or a combination thereof, and a toll like receptor-9 (TLR9) targeting ligand. The molecule can also contain lenalidomide, or an analogue or derivative thereof. Also disclosed is a composition comprising the disclosed molecule in a pharmaceutically acceptable carrier. Also disclosed is a method for treating non-del(5q) meylodysplastic syndrome (MDS) in a subject by administering to the subject a therapeutically effective amount of the disclosed pharmaceutical composition.

Abstract: The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the COL1A1, TGF-?, and SMAD2/3 genes, and methods of using such dsRNA compositions to inhibit expression of COL1A1, TGF-?, and SMAD2/3.

Abstract: The invention relates to improved RNA compositions for use in therapeutic applications. The RNA compositions are particularly suited for use in human therapeutic application (e.g., in RNA therapeutics). The RNA compositions are made by improved processes, in particular, improved in vitro-transcription (IVT) processes. The invention also relates to methods for producing and purifying RNA (e.g, therapeutic RNAs), as well as methods for using the RNA compositions and therapeutic applications thereof.

Abstract: The subject invention provides a novel SELEX strategy for isolating cross-reactive aptamers that recognize a core structure of a small-molecule family and bind to each structurally-similar molecule in said family. The subject invention also provides methods, assays, and products for detecting small-molecule targets of the family in a sample in both clinical and field settings. Such method is based on an aptamer sensor that reports the presence of small-molecule targets via a sensitive colorimetric signal for naked-eye detection. The subject invention further provides exonuclease-based methods for generating structure-switching aptamers from fully folded aptamers and developing electrochemical aptamer-based (E-AB) sensors for rapid and sensitive detection of synthetic cathinones.

Abstract: A method of determining an amount of transcript in a living organism, the method requiring editing a genome of a living organism to include a DNA copy of a unique microRNA barcode downstream of a promoter of a transcript to be measured, such that expression of that transcript includes expression of said microRNA barcode. Then, one collects secretions from said living organism and measures a level of said transcript by measuring the amount of the unique microRNA barcode present in those secretions. Cells, tissue and animals containing such barcodes are also included.

Abstract: The present invention relates to the fields of medicine and immunology. In particular, it relates to novel antisense oligonucleotides that may be used in the treatment, prevention and/or delay of Leber congenital amaurosis.

Abstract: The invention related to improved RNAi constructs and uses thereof. The construct has a double stranded region of 19-49 nucleotides, preferably 25, 26, or 27 nucleotides, and preferable blunt-ended. The construct has selective minimal modifications to confer an optimal balance of biological activity, toxicity, stability, and target gene specificity. For example, the sense strand may be modified (e.g., one or both ends of the sense strand it/are modified by four 2?—O-mehtyl groups), such that the construct is not cleaved by Dicer or other RNAse III, and the entire length of the antisense strand is loaded into RISC. In addition, the antisense strand may also be modified by 2?—O-methyl group at the 2nd 5?-end nucleotide to greatly reduce off-target silencing. The constructs of the invention largely avoids the interferon response and sequence-independent apoptosis in mammalian cells, exhibits better serum stability, and enhance target specificity.

Abstract: The present invention provides methods of enhancing the efficacy and specificity of RNA silencing. The invention also provides compositions for mediating RNA silencing. In particular, the invention provides siRNAs, siRNA-like molecules, shRNAs, vectors and transgenes having improved specificity and efficacy in mediating silencing of a target gene. Therapeutic methods are also featured.

Abstract: The invention relates to compostions and methods for promoting cellular proliferation and de-differentiation of cells into stem cells to foster tissue regeneration. Specifically, the invention relates to transiently administering a microRNA (miR) or its mimic for promoting cardiomyocyte proliferation and cardiac regeneration.

Abstract: In certain aspects, provided herein are RNA complexes that inhibit Myeloid differentiation primary response gene 88 (MyD88) and/or Toll-like receptor 3 (TLR3) and are useful in the treatment of age-related macular degeneration (AMD). In certain aspects, provided herein are pharmaceutical compositions comprising such RNA complexes and methods of using such RNA complexes and pharmaceutical compositions.

Abstract: The present invention is related to a nucleic acid molecule capable of binding to human C5a, wherein the nucleic acid molecule comprises a central stretch of nucleotides, wherein the central stretch of nucleotides comprises a nucleotide sequence of 5? AUGn1GGUGKUn2n3RGGGHUGUKGGGn4Gn5CGACGCA 3? [SEQ ID NO: 61], wherein n1 is U or dU, n2 is G or dG, n3 is A or dA, n4 is U or dU, n5 is U or dU and G, A, U, C, H, K, and R are ribonucleotides, and dU, dG and dA are 2?-deoxyribonucleotides.

Abstract: The present invention provides synthetic DNA aptamers that bind a target explosive to allow detection of that explosive. In various embodiments, the synthetic DNA aptamers may include one or more aptamers selected from the group consisting of SEQ ID 1-6. The various synthetic DNA aptamers are sensitive to different explosives. The synthetic DNA aptamers provide an inexpensive, in situ means for testing for explosive, which may be used for both soil and water samples. This testing may include an assay method using the synthetic DNA aptamers or a biosensor linked to the synthetic DNA aptamers.

Abstract: The subject invention provides methods, assays and products for detecting small-molecules in a sample, in particular, in both clinical and field settings. The method for detecting a small-molecule target in a sample comprises providing a sample, contacting the sample with an aptamer-based sensor selective for the small-molecule target, and sensitively and rapidly detecting the small-molecule target in the sample. Specifically, the method utilizes EATR-amplified small-molecule sensors based on cooperative binding split aptamers (CBSAs).