Abstract: Retrotransposons, operating though human-specific neurological pathways, can contribute to environment, lifestyle, and/or age-related neurodegeneration by disrupting functional mitochondrial populations within neurons. The mitochondrial disruption can occur through a number of retrotransposon-induced mechanisms that can influence the efficient and accurate transcription and/or translation of mitochondrial genes encoded in the nuclear genome, operating primarily through epigenetic processes. Alu element-related conformational changes (both subtle and major) of the outer and inner mitochondrial membrane pores can restrict or prevent the normal translocation of proteins (i.e., TOMM and TIMM complexes), ultimately contributing to mitochondrial stress, mitophagy, inflammation, and neuron and glial cell death.

Abstract: The invention belongs to the field of research and development of genetic engineering antibody drugs, in particular, relates to a method for treating a tumor by administering Circ-CDH1. The Circ-CDH1 is a circular RNA Circ-CDH1 nucleic acid molecule or the protein Circ-CDH1-28 KD expressed by the circular RNA Circ-CDH1 nucleic acid molecule. In particular, a monoclonal antibody Anti-Circ-CDH1 is designed against Circ-CDH1-28 KD, which can specifically detect the content of a protein encoded by endogenous circular RNA Circ-CDH1, can remarkably inhibit invasion and metastasis of cells from tumors such as glioma, breast cancer and the like, and has wide application prospects in clinical detection of tumors and invasion and metastasis treatment.

Abstract: In some aspects, the invention provides a method of treating atherosclerosis in a subject. The method comprises administering to the subject an agent that increases the activity or level of a let-7 miRNA or an agent that decreases activity or level of a TGF? signaling polypeptide in an endothelial cell in the subject. In some embodiments, the subject is administered an additional agent comprising a therapeutically effective amount of rapamycin or any derivative thereof. In some embodiments, the agent is a let-7 miRNA. In some other aspects, the invention provides a pharmaceutical composition comprising a let-7 miRNA. In some embodiments, the let-7 miRNA is encapsulated in a nanoparticle formulated for selective delivery to an endothelial cell.

Abstract: The present invention relates to methods of treating brain metastases using synergistic combinations of anti-PI3K and anti-mTOR therapies. It has also been determined that blocking PI3K and mTOR provides therapeutic benefit for treating brain metastases.

Abstract: The present invention relates to oligonucleotides that are capable of inducing expression of ubiquitin-protein ligase E3A (UBE3A) from the paternal allele in animal or human neurons. The oligonucleotides target the suppressor of the UBE3A paternal allele by hybridization to SNHG14 long non-coding RNA downstream of SNORD109B. The present invention further relates to pharmaceutical compositions and methods for treatment of Angelman syndrome.

Abstract: The invention relates to iRNA agents, which preferably include a monomer in which the ribose moiety has been replaced by a moiety other than ribose. The inclusion of such a monomer can allow for modulation of a property of the iRNA agent into which it is incorporated, e.g., by using the non-ribose moiety as a point to which a ligand or other entity, e.g., a lipophilic moiety. e.g., cholesterol, is is directly, or indirectly, tethered. The invention also relates to methods of making and using such modified iRNA agents.

Abstract: The purpose of the present invention is to provide a therapeutic agent which is for inflammatory bowel disease and exhibits superior prophylactic or therapeutic effects on inflammatory bowel disease by using miR-29a and/or miR-29b. The prophylactic or therapeutic agent for inflammatory bowel disease comprises composite particles in which miR-29a and/or miR-29b is/are carried on carbonate apatite particles, and said agent is administered systemically.

Abstract: The present invention provides iRNA agents, e.g., double stranded iRNA agents, that target the transthyretin (TTR) gene and methods of using such iRNA agents for treating or preventing TTR-associated diseases.

Abstract: This disclosure relates to oligonucleotides, compositions and methods useful for reducing LDHA expression, particularly in hepatocytes.

Abstract: Provided are methods and compositions useful in the diagnosis, treatment, and monitoring of osteosarcoma. Antisense to certain microRNA (miRNA) found to be associated with cancer stem cells (CSCs) or tumor-initiating cells (TICs) of osteosarcoma are useful to suppress tumor growth and metastasis, and prolong survival. Antisense oligonucleotides to miR-133a are synergistic in combination with standard chemotherapy such as cisplatin in the treatment of osteosarcoma.

Abstract: Compositions, devices, grafts and methods for reducing or preventing anti-neointima following cardiovascular injuries and interventions are disclosed. The compositions, devices, and grafts typically include an effective amount of a CTP synthase 1 inhibitor to reduce proliferation of vascular smooth muscle cells, without substantial reducing the proliferation of endothelial cells. Methods of reducing neointima formation, accelerating re-endothelialization, and reducing restenosis in a subject using the compositions, devices, and grafts are also disclosed.

Abstract: The presently-disclosed subject matter generally relates to RNAi-based methods and compositions for pest control of the highly invasive Emerald Ash Borer. The disclosed matter also relates to a composition which includes dsRNA directed towards target genes and a method of administering said composition.

Abstract: Provided herein are methods, compounds, and compositions for reducing expression of an ANGPTL3 mRNA and protein in an animal. Also provided herein are methods, compounds, and compositions for reducing plasma lipids, plasma glucose and atherosclerotic plaques in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate any one or more of cardiovascular disease or metabolic disease, or a symptom thereof.

Abstract: The present invention relates to compositions and methods for detecting motor neuron-specific miRNAs in a population of cells or subject. More particularly, the invention relates to detecting motor neuron-specific miRNAs to detect and treat motor neuron diseases associated with dysregulation of motor neuron-specific miRNAs, such as Amyotrophic Lateral Sclerosis (ALS).

Abstract: The present invention relates to antisense-oligonucleotides having a length of at least 10 nucleotides, wherein at least two of the nucleotides are LNAs, their use as inhibitors of TGF-R signaling, pharmaceutical compositions containing such antisense-oligonucleotides and the use for prophylaxis and treatment of neurological, neurodegenerative, fibrotic and hyperproliferative diseases.

Abstract: A method of modulating hygrosensing and/or thermosensing in an animal, particularly, an insect or disease vector, is provided. Also provided is a method of reducing survival, host-seeking, and/or reproductive capability of an animal, particularly an insect or disease vector. The methods involve an effective amount of an agent that modulates the activity and/or expression of a polynucleotide or polypeptide of an ionotropic receptor (Ir) selected from one or more of Ir25a, Ir93a, Ir40a, Ir68a, or Ir21a. A method of identifying an agent that modulates survival, host-seeking, and/or reproductive capability of an animal, e.g., an insect, is further provided.

Abstract: Provided is a drug that allows highly-efficient skipping of exon. The present invention provides an antisense oligomer wherein two or more unit oligomers targeting sequences that are neither consecutive nor overlap with each other in the same exon are connected.

Abstract: Provided herein are methods for using RNAi molecules targeting an Inhibitor of Apoptosis (IAP) gene for controlling Coleopteran insects, methods for producing RNAi molecules targeting IAP, and compositions comprising RNAi molecules targeting IAP.

Abstract: Provided are methods for the diagnosis and treatment of liver cancers such as hepatocellular carcinoma (HCC). In some aspects, the methods comprise administering an inhibitor of STEAP2 to a subject to treat a liver cancer. In some embodiments, a STEAP2 targeting siRNA or antibody is administered to a subject to treat HCC.

Abstract: The present invention provides an oligonucleotide comprising the sequence 5?-GGAACAGTTCGTCCATGGC-3? (SEQ ID NO:2) for use in the treatment of inflammatory bowel disease in a human subject, wherein individual doses of from 150 mg to 350 mg of said oligonucleotide are administered to the subject on at least two separate occasions, wherein said separate occasions are 3 weeks apart.