Abstract: Compositions and methods for modulating HIF-2? to meditate of hypoxia signaling in satellite cells and applications thereof for improving skeletal muscle generation and repair are provided. For example, methods of enhancing, increasing, accelerating or/and otherwise improving skeletal muscle generation or regeneration in a subject in need thereof are disclosed. In some embodiments, the methods include administering the subject an effective amount of HIF-2? inhibitor. The HIF-2? inhibitor can be effective to, for example, increase muscle satellite cell proliferation, differentiation, or a combination thereof in a subject. Composition and methods for improving respiration, and reducing or preventing the development or progression of fibrosis are also provided. The disclosed compositions and methods are particularly useful for treating muscular dystrophies, myopathies, and other muscle-related diseases and disorders.

Abstract: Loss of cardiomyocytes underlies most causes of heart failure, and normal repair processes are inadequate to deal with extensive myocardial damage. The inventors have identified mutations of the N-terminus of serum response factor (SRF)'s MADS box, termed STEMINs, that block cardiac differentiation, but also powerfully activate the stem cell marker genes Nanog and Octomer 4, as well as cyclins, which promotes adult myocyte replication. SRF Stemin mutations are not cardiac-specific, and also propel mammalian fibroblasts into a proliferative state. Thus, STEMINs may be useful for regeneration of all tissue and organ types, by activating partial pluripotency programs and enhancing repair by increased cell replication. Following withdrawal of STEMINs, the cells then return to normal cell identity.

Abstract: The present invention relates to agents for use in the treatment of glioma, in particular astrocytoma WHO II° and III°, as well as IV° (glioblastoma), in a subject.

Abstract: The invention relates to a double-stranded ribonucleic acid (dsRNA) targeting a G-alpha q subunit (GNAQ) of a heterotrimeric G gene, and methods of using the dsRNA to inhibit expression of GNAQ.

Abstract: Methods for modulating the release and content of extracellular vesicles from target cells using electrical stimulation are provided. Compositions comprising extracellular vesicles or extracts of extracellular vesicles and methods of administering the compositions to a subject are also provided.

Abstract: The present disclosure provides methods of treating subjects having a liver disease, and methods of identifying subjects having an increased risk of developing liver disease.

Abstract: Inhibitory RNA molecules that specifically inhibit mammalian RIZ2 expression, with therapeutic effect in cell proliferative diseases, such as cancer.

Abstract: A nucleic acid complex includes a single-stranded nucleic acid and a cross-linked double-stranded nucleic acid including the first nucleic acid strand linked to at least one of the 5? end and the 3? end of the single-stranded nucleic acid and the second nucleic acid strand including a base sequence that is completely or sufficiently complementary to the first nucleic acid strand.

Abstract: The present invention is based, in part, on methods for modulating regulatory T cells, regulatory B cells, and immune responses using modulators of the APRIL-TACI interaction.

Abstract: Disclosed herein include methods and compositions for nucleic acid synthesis using a terminal deoxynucleotidyl transferase with deoxyribonucleotide trisphosphates each comprising a modified base with a photocleavable carbon chain moiety that enables single incorporations when present.

Abstract: The invention provides compositions and methods for modifying the 3?-terminal ends of nucleic acids using DNA polymerase ? terminal transferase activity.

Abstract: Compositions of matter comprising RNA silencing molecules capable of mediating cleavage of p21 mRNA are disclosed. Methods of eradicating senescent cells or cancer cells, as well as methods of treating senescence-associated diseases or disorders, cancer, and fibrotic diseases and disorders are also disclosed.

Abstract: The present disclosure provides compositions and methods for targeted insertion of a gene of interest in the genome of a cell using single-stranded DNA or double-stranded DNA with 3 overhang. Also provided are methods of generating single-stranded DNA or double-stranded DNA with 3? over-hang that can be used for targeted insertion.

Abstract: The present disclosure provides methods of treating a subject having metabolic disorders and/or cardiovascular diseases, methods of identifying subjects having an increased risk of developing a metabolic disorder and/or a cardiovascular disease, and methods of detecting human Inhibin Subunit Beta E variant nucleic acid molecules and variant polypeptides.

Abstract: Provided herein are methods for miRNA profiling for the diagnosis, prognosis, and management of melanoma and differentiation of melanoma from nevi.

Abstract: Nucleotide sequences including a micro-dystrophin gene are provided. The micro-dystrophin genes may be operatively linked to a regulatory cassette. Methods of treating a subject having, or at risk of developing, muscular dystrophy, sarcopenia, heart disease, or cachexia are also provided. The methods may include administering a pharmaceutical composition including the micro-dystrophin gene and a delivery vehicle to a subject. Further, the methods may include administering the pharmaceutical composition a subject having Duchenne muscular dystrophy or Becker muscular dystrophy.

Abstract: Generally, the inventive technology relates to novel strategies for disease control in animal systems. Specifically, the inventive technology relates to novel methods, systems and compositions for the biocontrol of pathogens in aquatic systems. Specifically, the invention may comprise novel techniques, systems, and methods for the biocontrol of disease-transmitting pathogens affecting shrimp in aquaculture systems.

Abstract: The present invention provides non-propagating transgenic microalgae expressing at least one heterologous RNAi molecule. The RNAi-expressing non-propagating transgenic microalgae are used for oral delivery of the RNAi molecule to a target organism in its intact and functional form. The heterologous RNAi molecule, present within the microalgae, is characterized by being biologically active, exerting at least one specific effect on the organism consuming the microalgae or on a pathogen of said organism. In particular, the non-propagating transgenic microalgae are used as agents for biological control of animal and plant pests.

Abstract: The present invention relates to antisense RNAs targeting PMP22 and able to inhibit from 40% to 60% the expression of PMP22 in the cells and a pharmaceutical composition comprising thereof. The antisense RNAs are preferably siRNA and are preferably provided in the form of nanoparticles. The present invention also relates to the use of these antisense RNAs targeting PMP22 for the treatment of Charcot-Marie-Tooth 1A disease.

Abstract: The syntheses of two phosphoramidite building blocks based on BNSF and BNSMB structures are disclosed. Furthermore, some common molecular intermediates have been designed and linked to the central biphenyl core of the two molecules, resulting in a versatile and cost-effective design. These compounds can be effectively introduced to DNA oligonucleotides via the well-established standard cyanoethylphosphoramidite chemistry on the nucleic acid synthesizer. Fragmentation of these BNSF- and BNSMB-functionalized DNA strands is achieved by both one-photon and two-photon photolysis of photoliable bonds of [2-(2-nitrophenyl)propoxy]carbonyl groups on BNSF and BNSMB molecules respectively, resulting in two short pieces of single-stranded DNAs.