Abstract: An isolated or purified antisense oligomer for modifying pre-mRNA splicing in the CNOT3 gene transcript or part thereof.

Abstract: A composition useful for alleviating or treating pain and uses thereof are disclosed. The composition containing glutamate decarboxylase and a gene coding for an anti-inflammatory cytokine. A method for alleviating or treating pain of a subject includes administering the composition to the subject.

Abstract: The present invention relates to regulatory nucleic acid sequences, in particular liver-specific cis-regulatory elements, cis-regulatory modules, promoters and other such nucleic acid sequences, that are capable of enhancing liver-specific expression of genes. The invention also relates to expression constructs, vectors and cells comprising such liver-specific regulatory nucleic acid sequences, and to methods of their use. The liver-specific regulatory nucleic acid sequences are of particular utility for gene therapy applications, but also find utility in other areas such as bioprocessing and biotechnology.

Abstract: The present invention describes a novel bacterial nuclease of a CRISPR-Cas9 system from the bacterium P. pneumotropica, as well as the use of said nuclease for creating strictly specific two-strand cuts in a DNA molecule. The present nuclease possesses unusual properties and can be used as an instrument for introducing changes at strictly specified locations in a genomic DNA sequence of single-celled and multi-celled organisms. The invention thus increases the universality of accessible CRISPR-Cas9 systems, making it possible to use Cas9 nuclease from various organisms to cut genomic or plasmid DNA in a large number of specific sites and under various conditions.

Abstract: Provided is a promoter having high activity in an activated T-cell. The promoter comprises, from 5?-end to 3?-end, a CMV enhancer, an IFN? promoter, and a long terminal repeat sequence from human T-cell leukemia virus that are connected in sequence. The promoter exhibits greater activity in an activated immune cell than the existing promoters and is low in activity or inactive in other non-immune cells.

Abstract: A method of generating a cell that enhances functional myelin production is provided, the method including genetically modifying the cell such that: (i) an endogenous PLP1 gene is modified to decrease its ability to inhibit myelin production; (ii) an endogenous PLP1 genetic regulatory element is modified to decrease its ability to promote PLP1 expression; (iii) an endogenous PLP1 genetic regulatory element is modified to increase its ability to inhibit PLP1 expression; or (iv) an endogenous PLP1 gene product or a PLP1 regulatory element gene product that promotes PLP1 expression is modified to decrease the PLP1 expression level, wherein the cell produces functional myelin.

Abstract: The invention provides a nucleic acid comprising a Cpf1 crRNA, a processing sequence 5? of the Cpf1 crRNA, and an extension sequence 5? of the processing sequence. The invention also provides a composition comprising the nucleic acid, a carrier, and optionally Cpf1. Additionally, the invention provides method of genetically modifying a eukaryotic target cell, comprising contacting the eukaryotic target cell with the nucleic acid or the composition to genetically modify a target nucleic acid in the cell.

Abstract: The present invention relates to new aptamer molecules for use in therapy of infections caused by viruses from the Coronaviridae family, a method of preventing infection caused by viruses from the Coronaviridae family in vitro/ex vivo, a pharmaceutical composition and a kit comprising such aptamer molecules, and the use of aptamer molecules for preventing infection of somatic cells with a virus from the Coronaviridae family. The present invention also relates to affinity molecules binding to specific and newly identified epitopes of a key enzyme of Coronaviridae viruses.

Abstract: One aspect of the present invention relates to double-stranded RNA (dsRNA) agent capable of inhibiting the expression of a target gene. Other aspects of the invention relate to pharmaceutical compositions comprising these dsRNA molecules suitable for therapeutic use, and methods of inhibiting the expression of a target gene by administering these dsRNA molecules, e.g., for the treatment of various disease conditions.

Abstract: The disclosure relates to fusion proteins comprising a tBID polypeptide and a steroid hormone receptor domain, and methods of using same to induce apoptosis in cells.

Abstract: Disclosed are methods of treating perianal fistula a subject by administering a therapeutic MSC secretome product made by a method comprising culturing bone marrow-derived MSCs under conditions that include oxygen tension below 5% and a culture media with a pH below 7.

Abstract: A composition of matter includes an antisense phosphorodiamidate morpholino oligomer (MO) that includes an MO base sequence. The MO base sequence is arranged to bind a corresponding complementary base sequence of messenger RNA (mRNA) transcribed from one or more genes for the beta subunit of human chorionic gonadotropin (hCG-?). An inventive method, for suppressing intracellular synthesis of a beta subunit of human chorionic gonadotropin (hCG-?), includes introducing such an MO into one or more cells.

Abstract: An siRNA which inhibits kininogen (KNG) gene expression, a pharmaceutical composition containing the siRNA, and an siRNA conjugate. Each nucleotide in the siRNA is independently a modified or unmodified nucleotide. The siRNA contains a sense strand and an antisense strand. The sense strand contains nucleotide sequence I, nucleotide sequence I having the same length as the nucleotide sequence shown in SEQ ID NO: 1, with no more than three nucleotide differences. The antisense strand contains nucleotide sequence II, nucleotide sequence II having the same length as the nucleotide sequence shown in SEQ ID NO: 2, with no more than three nucleotide differences. The siRNA, the pharmaceutical composition thereof and the siRNA conjugate can effectively treat and/or prevent septicemia.

Abstract: Several embodiments relate to methods of repairing and/or regenerating damaged or diseased tissue comprising administering to the damaged or diseased tissues compositions comprising exosomes. In several embodiments, the exosomes comprise one or more microRNA that result in alterations in gene or protein expression, which in turn result in improved cell or tissue viability and/or function.

Abstract: Disclosed herein are compositions comprising a mesenchymal stem cell (MSC) preparation and one or more biomolecules and the use of said compositions for the treatment or a microbial infection or the symptoms or secondary pathological conditions associated with said infection.

Abstract: This application relates to potent oligonucleotides useful for reducing HBsAg expression and treating HBV infections.

Abstract: The present invention is directed to genome editing systems, reagents and methods for the treatment of hemoglobinopathies.

Abstract: An isolated or purified antisense oligomer for modifying pre-mRNA splicing in the CNOT3 gene transcript or part thereof.

Abstract: Aspects of the disclosure relate to compositions and methods for multiplexed gene silencing in a cell or subject. In some embodiments, the disclosure provides an isolated nucleic acid or an rAAV encoding a transgene comprising a RNA-guided nuclease (RGN) operably linked to a first promoter, and a second promoter operably linked to a multi guide-RNA (multi-gRNA) expression cassette encoding one or more gRNAs targeting a gene associated with hypercholesterolemia or dyslipidemia. In some embodiments, the disclosure provides methods of treating a subject having hypercholesterolemia or dyslipidemia by administering the compositions.

Abstract: The present invention relates to RNAi agents, e.g., dsRNA agents, targeting the transmembrane serine protein 2 (TMPRSS2) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of a TMPRSS2 gene and to methods of treating or preventing a TMPRSS2-associated disease, e.g., COVID-19, in a subject.