The present invention relates to a system and process for the production of a hairpin loop ended self-complementary double-stranded covalently closed linear deoxyribonucleic acid (DNA) vector, and the use of said vector as part of a therapeutic formulation enabling modulation of gene expression for medical applications. This system comprises a recombinant cell and at least two engineered parental circular covalently closed synthetic plasmids DNA, housed in the recombinant cell, wherein the synthetic transcriptional units of the at least two engineered parental circular covalently closed synthetic plasmids DNA are cloned in opposite directions. This DNA vector manufacturing system provides an efficient and high yield inducible process for producing hairpin loop ended linear covalently closed DNA vectors that incorporate a nucleic acid sequence of interest.

The present invention relates to multispecific transthyretin (TTR) complexes useful as multispecific binding proteins. The multispecific TTR complexes described herein are particularly useful in binding to one, two, or more epitopes which may be present on one or more proteins. Methods for treating diseases using the TTR complexes of the present invention are described herein.

Provided are an immune effector cell for co-expressing a chemokine receptor, a pharmaceutical composition, a kit, and a method for treating a tumor. The immune effector cell comprises a receptor that specifically recognizes claudin 18.2 and a protein that recognizes SDF-1. Further provided are an expression construct, an expression vector, and a virus. The expression construct comprises an expression of a receptor that binds to a tumor-associated antigen and an expression of the protein that recognizes SDF-1, which are connected in sequence.

The present disclosure provides methods of inducing EBV early lytic cycle genes with high specificity. These methods slow or stop cancer cell growth in vitro and in vivo.

The present invention relates to a composition for screening various signal peptides to select specific ones that allow efficient secretion of a target protein to out of host cells. The present invention also relates to a method for selecting specific signal peptides that express a target protein in host cells and efficiently secrete the target protein to out of the host cells. The use of the composition and/or method according to the present invention enables ultrafast selection of optimal signal peptides for a target protein through barcoding sequences corresponding to the signal peptides, leading to the maximization of the production yield of the recombinant protein.

The present disclosure relates to a pair of flanking sequences for a split intein, wherein the pair of flanking sequences includes: a flanking sequence a and a flanking sequence b; the flanking sequence a is located at the N-terminus of the split intein N-terminal protein splicing region (In), and is between the N-terminal extein (En) and the In; the flanking sequence b is located at the C-terminus of the split intein C-terminal protein splicing region (Ic), and is between the Ic and the C-terminal extein (Ec); and the split intein is selected from the group consisting of SspDnaE, SspDnaB, MxeGyrA, MjaTFIIB, PhoVMA, TVoVMA, Gp41-1, Gp41-8, IMPDH-1 and PhoRadA.

The present disclosure provides compositions and methods for use in genome engineering of induced pluripotent stem cells (iPSCs). Specifically, the methods and compositions described are useful for introducing transgenes into iPSCs such as pluripotent hematopoietic stem cells and/or progenitor cells (HSC/PC) using an CRISPR nuclease-based system (e.g., MAD7 nuclease-based system) and preparing immune-effector cells derived from the iPSCs.

This disclosure provides a monoclonal population of highly sialylated multimeric binding molecules where the population includes IgM antibodies, IgM-like antibodies, or other IgM-derived binding molecules, where the population of binding molecules has a higher level of sialic acid content than is found in normal serum IgM. Also provided are methods of producing such monoclonal populations of highly sialylated multimeric binding molecules.

A protein for visual color recognition and the like, the protein having a channel activity and including an amino acid residue different from the amino acid residue present in a first amino acid sequence represented by SEQ ID NO: 1, at a position or positions corresponding to one or two or more selections from the group consisting of the following positions in the first amino acid sequence: positions 53, 83, 87, 117, 120, 124, 137, 139, 142, 143, 146, 150, 169, 173, 177, 198, 204, 216, 217, 218, 231, 238, 245, and 247.

The present application relates to protease substrates, peptide sequences cleavable by a protease, polypeptides comprising a protease cleavage sequence and methods for production thereof, pharmaceutical compositions comprising a polypeptide comprising a protease cleavage sequence, and methods for releasing an antigen-binding domain or a ligand by the cleavage of a protease cleavage sequence included in a polypeptide.