Described herein is an intronic recognition element for splicing modifier (iREMS) that can be recognized by a small molecule splicing modifier compound of Formula (I) provided herein or a form thereof, wherein W, X, A and B are as defined herein. In one aspect, methods for modifying RNA splicing to modulate the amount of a product of a gene, wherein a precursor RNA transcript transcribed from the gene that contains an intronic REMS is modified utilizing a splicing modifier compound of Formula (I), are described herein. In another aspect, methods for modifying RNA splicing to modulate the amount of an RNA transcript or protein product encoded by a gene, wherein a precursor RNA transcript transcribed from the gene is modified to comprise an intronic REMS utilizing a splicing modifier compound of Formula (I), are described herein.

The present disclosure provides small hairpin nucleic acid molecules capable of stimulating interferon production. The nucleic acid molecules of the present disclosure has a double-stranded section of less than 19 base pairs and at least one blunt end. In certain embodiments, the molecule comprises at least one 5′-triphosphate and/or at least one 5′-diphosphate. In certain embodiments, compounds and/or compositions of the disclosure are useful for treating, ameliorating, and/or preventing SARS-CoV-2 viral infection, and/or ameliorating, minimizing, reversing, and/or preventing persistent SARS-CoV-2 viral infection, and/or minimizing or preventing SARS-CoV-2 viral infection-derived mortality and/or lethality, in a subject. In certain embodiments, compounds and/or compositions of the disclosure are useful for treating, ameliorating, and/or preventing SARS-CoV-2 viral infection in a tumor-bearing subject. In certain embodiments, compounds and/or compositions of the disclosure are useful for treating, ameliorating, and/or preventing SARS-CoV-2 viral infection in an immune-compromised and/or immunodeficient subject.

The present application relates to induction of a Treg phenotype in mammalian naïve CD4+ T cells. In certain embodiments, the methods and compositions described can be applied as methods to treat autoimmune disorders or transplant complications (e.g., lupus and graft-versus-host disease) and may be used in combination with, but do not require, systemic immune suppression, such as a chemotherapeutic agent. In particular, embodiments of the disclosure can utilize transforming growth factor-beta 2 (TGFB2), molecules that stimulate the production of TGFB2, inhibitors of molecules that suppress production of TGFB2, or molecules that effect the function of TGFB2 to induce a Treg phenotype in naïve CD4+ T cells from a mammal. Provided herein are embodiments and examples demonstrating the production of Treg cells, as well as the application of Treg cells in modulating the inflammatory response present in certain diseases.

Genomic safe harbors (GSH) for genetic therapies in human stem cells and engineered nanoparticles to provide targeted genetic therapies are described. The GSH and/or associated nanoparticles can be used to safely and efficiently treat a variety of genetic, infectious, and malignant diseases.

Described herein are aptamers capable of binding to human retinoic acid-inducible gene I protein (RIG-I); compositions comprising a RIG-I binding aptamer with a RIG-I; and methods of making and using the same.

The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the mannan binding lectin serine peptidase 2 gene (MASP2). The invention also relates to methods of using such RNAi agents to inhibit expression of a MASP2 gene and to methods of preventing and treating a MASP2-associated disorders, e.g., arthritis, IgA nephropathy, thrombotic microangiopathy, diabetic nephropathy and membranous nephropathy.

Provided herein are compositions and methods for decreasing tau mRNA and protein expression. These compositions and methods are useful in treating tau-related diseases and disorders.

The invention is directed to methods of treating TNBC in a patient by administering to the patient an agent that inhibits the expression or activity of cyclin-dependent kinase 19 (CDK19). In some embodiments, the agent may be a small molecule inhibitor, a polynucleotide (e.g., shRNA. siRNA), or a protein (e.g., an antibody). In some embodiments, the agent does not inhibit the activity or expression of CDK8.

The present invention relates to a nucleic acid molecule simultaneously inhibiting the expression of mTOR gene and STAT3 gene, and an anticancer pharmaceutical composition comprising the same. More specifically, base-paired siRNA or shRNA of the present invention, designed to simultaneously inhibit the expression of cancer-related mTOR gene and STAT3 gene in order to surmount the problem that siRNA or shRNA does not achieve high therapeutic effects due to the target specificity thereof, has the effect of promoting the death of cancer cells. In addition, the nucleic acid has the effect of synergistically enhancing the apoptosis of cancer cells when used in combination with an anticancer agent, finding useful applications as an anticancer composition or anticancer aid against various carcinomas.

Clean version of Abstract Small interfering RNA (siRNA) for inhibiting the expression of the mini-chromosome maintenance 7 (MCM7) gene, and a composition and use thereof are provided. The siRNA designed and verified by the present disclosure can effectively inhibit the expression of the MCM7 gene, and thus inhibit the DNA synthesis, cell proliferation, and colony formation of cancer cells, thereby achieving the purpose of preventing and treating a tumor. The present disclosure provides a new target and candidate compound for cancer prevention or treatment.