This disclosure provides, among other things, amplifiable nucleic acid constructs for expressing a gene of interest in a cell, e.g., an erythroid cell. The amplifiable nucleic acid construct may contain the gene of interest and an RNA-dependent RNA polymerase (RdRP)-responsive 5′ UTR, and may optionally further contain an RdRP-responsive 3′ UTR. RdRP may also be provided, e.g., on the same construct or a different construct.

A system and method for modulating stress granule assembly, utilizing a protein construct that has a cell penetrating protein fused to one or more proteins that can bind with an NTF2-like domain of a G3BP protein. By configuring the protein construct with an appropriate number of proteins that being with NTF2-like domains, stress granule assembly can be upregulated or downregulated as needed to treat patients.

Methods for increasing targeted genome editing by down-regulating proteins involved in cytosolic DNA sensing pathways.

Methods and compositions of altering a eukaryotic cell are described including providing to the eukaryotic cell a guide DNA sequence complementary to a target nucleic acid sequence, providing to the eukaryotic cell an Ago enzyme or a nuclease null Ago protein that interacts with the guide DNA sequence for DNA-guided gene editing and regulation of the target nucleic acid sequence in a site specific manner.

The present invention includes a method, kits, and assays for identifying a human subject as having an increased risk of developing an autoimmune disease, or a human subject with multiple sclerosis caused by elevated soluble Interleukin 7 receptor (sIL7R), by obtaining a biological sample and detecting or measuring in the biological sample an amount of a soluble Interleukin-7 receptor (sIL7R) and an amount of an RNA Helicase DDX39B, whereby a lower expression of DDX39B and a higher secretion of sIL7R identifies the subject from which the biological sample was obtained as having an increased risk of developing an autoimmune disease, when compared to a human subject not having an autoimmune disease. The present invention also includes a method of modifying a treating of subjects based on the lower expression of RNA Helicase DDX39B alone or in combination with an increase in sIL7R.

A nucleic acid molecule encoding a fusion protein composed of an inducible multimerization moiety at the amino terminus, and a GTPase-Activating Protein SH3 Domain-Binding Protein (G3BP) is provided for exogenous stimulus/G3BP-mediated stress granule formation. Further disclosed are chemical or light inducible multimerization proteins or protein domains that can be used for the nucleic acid molecule production, and a method of inducing stress granule formation in a cell comprising expressing the nucleic acid molecule.

Disclosed is a method for prophylaxis or treatment of infection of a virus, or for modulating innate immunity, in a subject comprising administering to the subject a therapeutically effective amount of a PUM1 inhibitor. A pharmaceutical composition comprising the PUM1 inhibitor is also disclosed.

Provided are DDX24 mutations and the use thereof, wherein the mutations include Glu271Lys, Lys11Glu and Arg436His. The mutations, i.e. Glu271Lys, Lys11Glu and Arg436His, of DDX24 gene are significantly associated with the development of blood vessels. It would result in vascular malformations by interfering with DDX24. In addition, the onset of human vascular malformations can be predicted by detecting the SNP sites of DDX24.

The invention relates to a method for preventing, ameliorating or treating disease caused by dengue virus in a subject in need thereof comprising administering to the subject a dengue vaccine formulation in combination with a NS3 helicase polypeptide and/or fragment(s) thereof, wherein said method comprises stimulating humoral as well as cell-mediated immunity to the dengue virus in the subject.

The present disclosure provides a promoter having at least the core components of a duck retinoic acid-inducible gene I (RIG-I) promoter, as well as expression constructs having the duck RIG-I promoter operably linked to a gene product-encoding nucleic acid (e.g., an avian RIG-I protein), and recombinant host cells containing the duck RIG-I promoter, e.g., in such expression constructs. The present disclosure also provide animals genetically modified to have a gene encoding a duck RIG-I promoter operably linked to a gene product-encoding nucleic acid (e.g., an avian RIG-I protein, such as a duck RIG-I protein).