Abstract: Certain disclosed oligomers induce exon skipping during processing of myostatin pre-mRNA. The oligomers may be in a vector or encoded by the vector. The vector is used for inducing exon skipping during processing of myostatin pre-mRNA. A therapeutically effective amount of the oligomer may be administered to a subject patient such that exon skipping during processing of myostatin pre-mRNA is induced. The administration to a subject may be used in order to increase or maintain muscle mass, or slowing degeneration of muscle mass in the subject. The administration to a subject may ameliorate muscle wasting conditions, such as muscular dystrophy. Examples of such muscular dystrophies which may be so treated include Becker's muscular dystrophy, congenital muscular dystrophy, Duchenne muscular dystrophy, distal muscular dystrophy, Emery-Dreifuss muscular dystrophy, facioscapulohumeral muscular dystrophy (FSHD), limb-girdle muscular dystrophy, myotonic muscular dystrophy, and oculopharyngeal muscular dystrophy.

Abstract: The present disclosure relates to a method of delivering target materials into extracellular vesicles including exposing the target materials and the extracellular vesicles to extracorporeal shockwaves, a method of preparing target material-introduced extracellular vesicles, extracellular vesicles prepared by the method, drug delivery vehicles including extracellular vesicles, and a method of delivering target materials into cells. The present disclosure exposes extracellular vesicles derived from natural organisms such as animal cells, plant cells, and microorganisms including bacteria and eukaryotic bacteria as well as artificially produced extracellular vesicles to extracorporeal shockwaves extracellularly. Thus, the high-level energy extracorporeal shockwaves can be used to deliver the target material into the extracellular vesicle efficiently. When treating with extracorporeal shockwaves, the ability of target material-introduced extracellular vesicles to incorporate into target cells also increases.

Abstract: Disclosed herein is a method for diagnosing a renal allograft recipient's risk for developing fibrosis of the allograft and allograft loss. The method includes determining the expression levels of certain microRNAs, which have been determined to be predictive of an allograft recipient's risk. Also disclosed herein is a method of treating a renal allograft recipient to inhibit fibrosis of the allograft and allograft loss, as well as kits for use in the methods disclosed herein.

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 (HD) progression using an antisense compound targeted to huntingtin. Additionally provided are methods of delaying or preventing the onset of Huntington's Disease (HD) in an individual susceptible to Huntington's Disease (HD). Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders.

Abstract: Provided is a use of one or more MicroRNA genes selected from miRNAs of Family Let-7, miR-21 or miR-222 in the construction of tissue engineered nerves and in the repair of peripheral nerve defects. An outer and/or internal surface or pores of a tissue engineered nerve graft are coated or adsorbed with polymeric nanomicrospheres carrying a Let-7 family miRNA inhibitor, miR-21, or miR-222, or a mimetic thereof, wherein the polymeric material is composed of biocompatible fibronectin and heparin. The regeneration of peripheral nerves and the construction of tissue engineered nerves are promoted by regulating the expression of MicroRNA genes which can effectively promote the proliferation of primary Schwann cells cultured in vitro and have an anti-apoptotic effect on neuronal cells. In-vivo test proves that bridging of the tissue engineered nerve graft can facilitate the regeneration of peripheral nerves, thus being useful in the treatment of peripheral nerve injury.

Abstract: The present invention provides a screening method for pain suppressors, which method is characterized by using netrin-4 and/or a netrin-4 receptor to select a substance capable of inhibiting downstream signaling from netrin-4. According to the screening method of the present invention, pain suppressors useful as a preventive or therapeutic medicine for pain can be identified. The present invention also provides a pharmaceutical composition for prevention or treatment of pain, which composition comprises, as an active ingredient, a substance capable of inhibiting downstream signaling from netrin-4.

Abstract: Disclosed herein are methods and compositions useful in targeting a payload to, or editing a target nucleic acid, where a governing gRNA molecule is used to target, optionally inactivate, a Cas9 molecule or a Cas9 molecule/gRNA complex.

Abstract: The invention relates to antisense oligonucleotidic sequences (ODN) against Smad7 suitably modified, and their uses in medical field as therapeutic biological agents, in particular in the treatment of chronic inflammatory bowel disease, such as Crohn's disease and ulcerative colitis.

Abstract: The methods and assays described herein relate to detection, diagnosis, and treatment of lung cancer, e.g., by detecting the level of expression of certain miRNAs described herein and/or by therapeutically increasing the level of those miRNAs.

Abstract: The present invention features compositions and methods relating to tRNA-derived small RNAs (tsRNAs). Provided herein are oligonucleotide compositions that are complementary to tsRNAs, in particular leuCAGtsRNA, and methods of using the oligonucleotides for the regulation of respective tsRNA. Further provided are methods of inducing apoptosis through the inhibition of leuCAGtsRNA.

Abstract: The invention relates to non-CpG single-stranded oligonucleotides (ssONs) for use in the treatment or prophylaxis of disorders of the skin and/or subcutaneous tissue, including pruritus, in a suitable formulation or in combination with other immunomodulatory treatments. The said ssONs have a length of at least 25 nucleotides and are stabilized by phosphorothioate internucleotide linkages and/or 2?-O-Methyl modifications.

Abstract: Provided herein are methods, compounds, and compositions for reducing expression of huntingtin mRNA and protein in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate Huntington's disease, or a symptom thereof.

Abstract: In certain embodiments, methods, compounds, and compositions for treating B-cell lymphoma or hepatocellular carcinoma by inhibiting expression of STAT3 mRNA or protein in an animal are provided herein. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate B-cell lymphoma or hepatocellular carcinoma.

Abstract: Methods of treating an age-related disorder in a subject are provided. Aspects of the methods include administering to the subject a nucleic acid vector including a coding sequence for telomerase reverse transcriptase (TERT) and/or telomerase RNA (TR). Gene therapy methods are also provided. Aspects of the invention further include compositions, e.g., nucleic acid vectors and kits, etc., that find use in methods of the invention.

Abstract: Chronic inflammation is an increasing medical problem area of high socioeconomic significance. The invention relates to a method and a kit for diagnosing a molecular phenotype of a patient suffering from an illness accompanied by chronic inflammation, and to a medicament for treating such a patient. To that end, the gene expression of GATA-3 and/or Tbet in a biological isolate of the patient is measured and used for association with a molecular phenotype of the illness.

Abstract: The presently-disclosed subject matter relates to an artificial RNA nanostructure molecule and method to treat brain tumor in a subject. More particularly, the presently disclosed subject matter relates to a RNA nanostructure containing a multiple branched RNA nanoparticle, a brain tumor targeting module, and an effective amount of a therapeutic agent. Further, the presently disclosed subject matter relates to a method of using the RNA nanostructure composition to treat brain tumor in a subject having or at risk of having brain tumor.

Abstract: The invention relates to the use of an antisense compound for inducing exon inclusion as a treatment for Spinal Muscle Atrophy (SMA). More particularly it relates to inducing inclusion of exon 7 to restore levels of Survival Motor Neuron (SMN) protein encoded by the Survival Motor Neuron (SMN) gene.

Abstract: Cell death is induced and/or cell growth is suppressed for a cell having a mutation in the BRAF gene. A drug suppressing GST-? is comprised as an active ingredient.

Abstract: Double-stranded modified siRNA targeting a RecQL1 helicase gene includes a sense strand including the nucleotide sequence shown in SEQ ID NO: 1, and an antisense strand including the nucleotide sequence shown in SEQ ID NO: 2, wherein the sense strand includes 2?-substituted nucleotides at positions 2, 3, 4 and 13 in the nucleotide sequence shown in SEQ ID NO: 1, the sense strand further includes a 2?-substituted nucleotide(s) at one or more positions selected from the group consisting of positions 12, 14, 17, 18 and 19 in the nucleotide sequence shown in SEQ ID NO: 1, wherein the position 2? of the 2?-substituted nucleotides is —R1, —OR1, —R2OR1, —OR2OR1 or —R3OR2OR1, wherein R1 represents a C1-4 alkyl group, and R2 and R3 independently represent a C1-3 alkylene group.

Abstract: The present disclosure provides pharmaceutical compositions comprising nucleic acids capable of targeting IGF-1R expression in M2 cells. The present disclosure also provides methods for the selective reduction of M2 cells by targeting expression of IGF-1R in these cells. The present disclosure further provides methods for treating cancer and enhancing therapeutic by targeting expression of IGF-1R in M2 cells in patients. The pharmaceutical composition of the present invention is effective when administered systemically to subjects in need thereof. The ease of administration of the pharmaceutical composition facilitates treatment and enhances patient compliance.