Abstract: Described are methods for utilizing X-aptamers for detecting human SNAP25 and fragments thereof, and compositions comprising the X-aptamers.

Abstract: A method of treating a subject suffering from cancer comprising a step of administering an effective amount of a group of double-stranded RNA molecules to the subject, wherein the RNA molecule is isolated or derived from a bacteria of the genus Escherichia. A method of inhibiting growth or proliferation of cancer cells comprising a step of contacting said cells with said RNA molecule; and a pharmaceutical composition for treating cancer comprising said RNA molecule and a pharmaceutically tolerable excipient. Also a double-stranded RNA molecule and a recombinant vector comprising the double-stranded RNA molecule.

Abstract: A DNA aptamer, a pharmaceutical composition comprising the above DNA aptamer, a method for inhibiting the catalytic ability of TXNDC5 and a method for preventing or treating organ fibrosis are revealed. The DNA aptamer comprises a polynucleotide specifically binding to TXNDC5, the polynucleotide is selected from the group consisting of the nucleotide sequence of any one of SEQ ID NOS: 1-14. The pharmaceutical composition comprises the above DNA aptamers as an active ingredient. The method for inhibiting the catalytic ability of TXNDC5 comprises binding the above aptamers to TXNDC5. The method for preventing or treating organ fibrosis comprises administering an effective amount of the above aptamers to a subject.

Abstract: The invention relates to antisense oligonucleotides that are capable of bringing about specific editing of a target nucleotide (adenosine) in a target RNA sequence in a eukaryotic cell, wherein said oligonucleotide does not, in itself, form an intramolecular hairpin or stem-loop structure, and wherein said oligonucleotide comprises a non-complementary nucleotide in a position opposite to the nucleotide to be edited in the target RNA sequence.

Abstract: RNA interference (RNAi) triggers and RNAi trigger conjugates for inhibiting the expression of Hif2? (EPAS1) gene are described. Pharmaceutical compositions comprising one or more Hif2? RNAi triggers optionally with one or more additional therapeutics are also described. Delivery of the described Hif2? RNAi triggers to tumor cells in vivo provides for inhibition of Hif2? gene expression and treatment of cancer.

Abstract: The present disclosure provides methods of treating patients having an ophthalmic condition, methods of identifying subjects having an increased risk of developing an ophthalmic condition, methods of detecting human Son of Sevenless 2 (SOS2) variant nucleic acid molecules and variant polypeptides.

Abstract: Alternative splicing events in genes can lead to non-productive mRNA transcripts which in turn can lead to aberrant protein expression, and therapeutic agents which can target the alternative splicing events in genes can modulate the expression level of functional proteins in patients and/or inhibit aberrant protein expression. Such therapeutic agents can be used to treat a condition or disease caused by protein deficiency and/or mitochondrial function deficit.

Abstract: The current invention includes systems, methods and compositions for the generation of sRNA molecules using select RNase III mutants. In one preferred embodiment, invention includes systems, methods and compositions for the generation of sRNA molecules using RNase III mutants to control a host pathogen through the production and diffusion of sRNA molecules that may initiate an RNAi pathway response directed to a host pathogen. Additional embodiments of the current invention include systems, methods and compositions for the DICER-independent generation of sRNA molecules using select RNase III mutants.

Abstract: Provided are LMNA-targeted antisense oligonucleotides for reducing expression of one or more aberrantly spliced LMNA mRNA isoforms that encode progerin.

Abstract: Compositions and methods for treating or inactivating viruses in a subject in need thereof are provided herein. Exemplary compositions include nucleic acids encoding an RNA-guided endonuclease and a guide RNA that is complementary to a target sequence in a virus. The RNA-guided endonuclease specifically targets viral nucleic acid sequences for destruction and suppression of that virus in a host cell in vitro or in vivo.

Abstract: A CRISPR/Cas system and method for editing or regulating transcription of a genome of a cell are provided, wherein the system includes a Cas endonuclease fused with one or more degron sequences and at least one activatable cognate single guide RNA harboring an inactivation sequence in a non-essential region of the cognate sgRNA, wherein said inactivation sequence comprises one or more endonuclease recognition sites of, e.g., a ribozyme.

Abstract: Disclosed is a substance which down-regulates the activity of a MAST gene, or the activity of a transcription or translation product of a MAST gene, for use in the prevent and/or treatment of an inflammatory skin condition in a mammalian subject.

Abstract: Polynucleotides encoding peptides, proteins, enzymes, and functional fragments thereof are disclosed. The polynucleotides of the disclosure can be effectively delivered to an organ, such as the lung, and expressed within cells of the organ. The polyribonucleotides of the disclosure can be used to treat a disease or condition associated with cilia maintenance and function, impaired function of the axoneme, such as DNAI1 or DNAH5.

Abstract: RNA editing is achieved using oligonucleotide constructs comprising (i) a targeting portion specific for a target nucleic acid sequence to be edited and (ii) a recruiting portion capable of binding and recruiting a nucleic acid editing entity naturally present in the cell. The nucleic acid editing entity, such as ADAR, is redirected to a preselected target site by means of the targeting portion, thereby promoting editing of preselected nucleotide residues in a region of the target RNA which corresponds to the targeting portion.

Abstract: The present disclosure relates to compositions, such as siRNA molecules and FN3 domains conjugated to the same, as well as methods of making and using the molecules.

Abstract: This invention relates to compounds, compositions, and methods useful for reducing C5 target RNA and protein levels via use of dsRNAs, e.g., Dicer substrate siRNA (DsiRNA) agents.

Abstract: Aspects of the disclosure relate to compositions and methods useful for treating Huntington's disease. In some embodiments, the disclosure provides interfering nucleic acids (e.g., artificial miRNAs) targeting the huntingtin gene (HTT) and methods of treating Huntington's disease using the same.

Abstract: Aspects of the disclosure relate to synthetic regulatory systems composed of a multifunctional Cas nuclease, at least two guide RNAs (gRNAs) configured to target distinct nucleotide sequences, and a multilayered regulatory control element comprising ribozyme-based safety switches providing spatial and temporal control over the synthetic circuit in vivo.

Abstract: Provided herein are, inter alia, oligonucleotides, kits, and methods useful for increasing lentiviral titers.

Abstract: Diabetic wounds have elevated levels of cathepsin K, a protease enzyme, which degrades proteins, such as collagen, causing delayed wound healing. By targeting cathepsin K, diabetic wound healing is ameliorated. Methods and devices for treatment include intradermal injection of odanacatib at or adjacent a wound. Methods and devices for treatment include topical application of pharmacological inhibitors of cathepsin K, for example, in the form of gel, powder, or bandage. Other methods and systems include localized genetic knock out of the cathepsin K gene by topical application of a small interfering RNA (siRNA) or antisense oligonucleotide to aid in wound healing.