C12N2800/107

Expression from transposon-based vectors and uses

Recombinant expression vectors are disclosed that include a control sequence for recombinant expression of proteins of interest; the control sequence combines a mCMV enhancer sequence with a rat EF-1alpha intron sequence. Some of the vectors are useful for tetracycline-inducible expression. Some of the vectors contain a 5′ PiggyBac ITR and a 3′ PiggyBac ITR to promote genomic integration into a host cell chromosome. A method of selecting a stable production cell line for manufacturing a protein of interest is also disclosed. Also disclosed are mammalian host cells comprising the inventive recombinant expression vectors and a method of producing a protein of interest, in vitro, involving the mammalian host cell.

ANTI-IL-4R SINGLE-DOMAIN ANTIBODY AND USE THEREOF
20220411519 · 2022-12-29 ·

Disclosed is an anti-IL-4R single-domain antibody and the use thereof. In particular, disclosed are a IL-4R single-domain antibody and a VHH chain thereof, a coding sequence encoding the above-mentioned single-domain antibody or the VHH chain thereof, a corresponding expression vector, host cells capable of expressing the single-domain antibody, and a method for producing the single-domain antibody. The single-domain antibody can specifically recognize human and marmoset IL-4R, but does not recognize mouse IL-4R, and the specificity is good; the single-domain antibody can effectively inhibit the proliferation of TF-1 cells and the activation of a pSTAT6 signaling pathway in cells.

REPORTER CONSTRUCTS, COMPOSITIONS COMPRISING THE SAME, AND METHODS OF USE THEREOF
20220403475 · 2022-12-22 ·

Described herein are nucleic acid constructs that can be used as a reporter along with compositions and systems comprising the same. Also described herein are methods of use of such constructs, compositions and/or systems.

CONSTRUCTS FOR IMPROVED HDR-DEPENDENT GENOMIC EDITING

The invention provides an improved genome editing construct which is capable of editing a target sequence in an HDR-dependent manner (i.e., “HDR-dependent genome editors”) with increased efficiency and reduced indel formation and which does not require a dividing cell. In particular, the instant specification provides a new fusion protein comprising a nucleic acid programmable DNA binding protein (napDNAbp) (e.g., Cas9) with a nickase activity and a single-stranded DNA binding protein (e.g., Rad51) which edits a target DNA in an HDR-dependent manner with greater efficiency (e.g., increased rate of induced HDR) and/or with a lower rate or occurrence of indel formation.

TRIPARTITE SYSTEMS FOR PROTEIN DIMERIZATION AND METHODS OF USE

The disclosure provides compositions and methods that make use of a target protein that is capable of binding to a small molecule in order to form a complex, and a binding member that specifically binds to the complex, wherein the target protein is derived from a non-human protein and the small molecule is an inhibitor of the non-human protein. The non-human protein may be derived from a viral, bacterial, fungal or protozoal protein. These compositions and methods permit the controlled interaction of polypeptides that are individually fused to the target protein and binding member, respectively, and can be used to control the activity of dimerization-inducible proteins such as split transcription factors and split chimeric antigen receptors through the addition of the small molecule. The disclosure provides expression vectors, binding members, dimerization-inducible proteins, nucleic acids, cells, viral particles, kits, systems and methods that involve these components.

ENGINEERED CHIMERIC FUSION PROTEIN COMPOSITIONS AND METHODS OF USE THEREOF
20220378824 · 2022-12-01 ·

The present disclosure provides compositions and methods for making and using engineered phagocytic cells that express a chimeric antigen receptor having an enhanced phagocytic activity for immunotherapy in cancer or infection.

Treatment of neuropathy with DNA constructs expressing IGF-1 isoforms
11510999 · 2022-11-29 · ·

The present invention relates to a DNA construct encoding one or more human IGF-1 isoforms that can be used for treatment of neuropathy. Further provided herein are a pharmaceutical composition including the DNA construct as an active ingredient and a method of administering the DNA construct for treatment of neuropathy. The present invention provides a safe and effective way of treating neuropathic patients.

SELECTIVE CELL DEATH-INDUCING ENZYME SYSTEM
20220370555 · 2022-11-24 · ·

The invention relates to a fusion protein containing a selective cell death-inducing enzyme system for use in the therapy and/or treatment of cancer and tumors in humans and animals, a process, and its use.

PROMOTER HAVING HIGH ACTIVITY IN ACTIVATED T-CELL

Provided is a promoter having high activity in an activated T-cell. The promoter comprises, from 5′-end to 3′-end, a CMV enhancer, an IFNγ promoter, and a long terminal repeat sequence from human T-cell leukemia virus that are connected in sequence. The promoter exhibits greater activity in an activated immune cell than the existing promoters and is low in activity or inactive in other non-immune cells.

RECOMBINANT SEQUENCE SPECIFICALLY EXPRESSING HUMAN BETA-GLOBIN IN ERYTHROID CELLS, AND USE THEREOF
20220372510 · 2022-11-24 ·

Disclosed is an erythroid-specific human β-globin gene promoter and a human β-globin-expressing recombinant sequence, and use thereof, which belongs to the technical field of genetic engineering. An erythroid-specific human β-globin gene promoter with a nucleotide sequence set forth in SEQ ID NO: 1 is provided in the present disclosure, which may achieve high-efficiency and erythroid-specific initiation of the expression of a functional gene. A recombinant sequence specifically expressing human β-globin in erythroid cells is also provided in the present disclosure, which is human β-globin locus control region (LCR) HS 3-1+β-globin gene promoter+β-globin gene+β-globin gene enhancer.