The compositions and methods described herein relate to lipid nanoparticle encapsulation of circular or linear DNA sequences, including DNA vectors, for delivery into a subject such that prolonged expression in vivo occurs. Lipid nanoparticles containing DNA can be administered to a subject to express therapeutic polypeptides.

The invention relates to the use of a virus, preferably an adenovirus, for producing a medicament. Said virus is replication-deficient in cells which do not contain YB-1 in the core and codes for an oncogene or oncogene product, especially an oncogene protein, which transactivates at least one viral gene, preferably an adenoviral gene, said gene being selected among the group comprising E1B55kDa, E4orf6, E4orf3, and E3ADP.

A recombinant expression vector comprising a nucleotide sequence encoding a herpesvirus transactivator, where the nucleotide sequence is operably linked to a herpesvirus control element is provided as are cell lines genetically modified to express a herpesvirus transactivator under the control of a herpesvirus control element. Also provided are methods of identifying agents that disrupt feedback regulation of a herpesvirus transcriptional control element by a herpesvirus transactivator.

Provided herein is a cell line with improved odorant receptor function comprising an activated endogenous RTP1 gene, which further expresses an RTP1 protein. Further provided herein is a method for specifically activating an endogenous RTP1 gene in a eukaryotic cell using a CRISPR/Cas9 derived technique. Also provided herein is a method for identifying compounds with desired effects such as perfume or aroma modulators in said cell line.

The present invention includes compositions and methods for an HLA-A2 specific chimeric antigen receptor (CAR). In certain embodiments the HLA-A2 specific CAR is expressed on a T regulatory cell. In certain embodiments, the HLA-A2 specific CAR protects transplanted tissue from rejection.

Suggested is a method for enhancing the expression of taste related receptor genes encompassing the following steps: (i) providing a culture of mammalian cells, the genome of said cells comprising at least one sweet receptor domain; (ii) designing at least one type of single-guide RNA (sgRNA), the 10 to 30 nt guide sequence of said sgRNA being complementary to stretches within the non-coding and/or putative regulatory region upstream of the translation start codon of at least one sweet receptor gene; (iii) preparing a vector comprising an expression cassette encompassing at least one optionally modified CRISPR-Cas9, preferably CRISPR-dCas9VP64, and at least one optionally modified sg-RNA optionally containing aptamer structures for binding activator proteins; (iv) transfecting said culture of mammalian cells with said vector to target the genome for the presence of a DNA sequence that is complementary to the 10 to 30 nt guide sequence of said sgRNA; and (v) measuring the transcriptional enhancement of the sweet receptor mRNA by quantitative RT-PCR.

Disclosed herein is a subgenomic promoter library derived from alphavirus. Also provided herein are methods of using the subgenomic promoters to produce antibodies and other molecules.

The present invention discloses a recombinant bacteriophage comprising a phage genome polynucleotide including a gene encoding a heterologous antigen protein(s) and a killing gene encoding a protein that is capable of killing a host bacterium. Such a recombinant bacteriophage is designed to prime a subject's immune response and to kill the bacterium that it infects such that the prime and kill bacteriophage provides two lines of protection against infectious disease.

The present invention provides constructs comprising modified riboswitches to regulate expression of a transgene within a subject. Methods of treating a disease, specifically an eye disease, are also contemplated.

Provided is a gene expression cassette for stably and highly producing a protein of interest. The gene expression cassette has a structure in which a DNA construct (X) containing a gene of interest and a poly A addition sequence is sandwiched between a promoter (P) and an enhancer (P), the gene expression cassette further including transposon sequences (T) upstream of the promoter (P) and downstream of the enhancer (P). Further, in the gene expression cassette, when a nuclear matrix binding sequence (M) is appropriately arranged upstream of a replication initiation sequence (S) in combination with the transposon sequence (T), the protein of interest can be more effectively produced stably and in a large amount. For example, HRG, PD-1, EMMPRIN, NPTN, EMB, RAGE, MCAM, ALCAM, ErbB2, and an antibody can each be produced stably and in a large amount.