Abstract: Provided herein are compositions that include a single nucleic acid vector or two different nucleic acid vectors, and the use of these compositions to treat hearing loss in a subject.

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

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

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 circular RNA platforms, their manufacturing processes from an engineered parental circular covalently closed synthetic plasmid DNA and, uses thereof. Novel engineered circular covalently closed plasmids comprising sequences capable of aiding in RNA circularization either autonomously or when processed with an engineered ligase are provided. The circular RNA platforms of the current invention have improved stability and increased half-life, display exceptional and stable protein production, while avoiding double-stranded intramolecular self-pairing RNA segments.

Abstract: The disclosure relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the UGT1a1 gene, and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of UGT1a1.

Abstract: An inducible promoter expression system based on the T7 RNA Polymerase (T7 RNAP), lactose repressor (Lad), and a chimeric T7lac promoter (PT7lac), which can be integrated as a single copy into the B. subtilis genome. In the absence of IPTG, Lad strongly represses T7RNAP and PT7lac, and expression of an exemplary ORF—here superfolder green fluorescent protein (sfGFP) reporter protein—is undetectable by flow cytometry. Addition of IPTG de-represses PT7lac, and simultaneously induces expression of T7RNAP, resulting in very high sfGFP levels.