Abstract: The present invention provides a mutated woodchuck post-transcriptional regulatory element (WPRE). In particular, the present invention relates to a mutated WPRE sequence that can efficiently express nucleotides of interest in a retroviral vector system. The present invention also relates to methods of delivering and expressing nucleotides of interest to a target cell.

Abstract: Provided is a method for preparing functional hepatic (progenitor) cells or functional small intestinal epithelial (progenitor) cells, comprising the step of culturing an isolated cell population comprising hepatic (progenitor) cells or small intestinal epithelial (progenitor) cells in the presence of an antibiotic. Also, provided is a substantially homogeneous isolated cell population comprising functional hepatic progenitor cells, wherein an expression level of CYP3A4 in the functional hepatic progenitor cells is increased by at least 5 times the expression level thereof in a HepaRG® cell line.

Abstract: The invention generally relates to methods of performing a capture reaction. In certain embodiments, the method involves obtaining a nucleic acid, fragmenting the nucleic acid, and capturing a target sequence on the nucleic acid fragment using a capture moiety, such as a molecular inversion probe.

Abstract: A method is provided for detecting one or more analytes in a sample. The method relies, in part, on the ability of functionalized particles added to the sample to partially or completely inhibit the transmission of electromagnetic radiation into and out of the sample through a detection surface in a reaction vessel containing the sample. In a microarray format, the invention can be used to screen millions, billions or more biological elements, such as an organism, cell, protein, nucleic acid, lipid, saccharide, metabolite, or small molecules. Methods, apparatuses and kits are described.

Abstract: This disclosure relates to the field of research and clinical usage of extracellular vesicles. More in particular, this disclosure relates to usages of recombinant extracellular vesicles comprising a) a self-assembling protein that directs its own release through vesicles as a luminal membrane-bound protein and b) a heterologous marker. Indeed, the disclosure provides recombinant extracellular vesicles that can be used as a biological reference material in methods to quantify extracellular vesicles in a sample, and/or, that can be used to calibrate a device for sample extracellular vesicles analysis, and/or, that can be used to evaluate the isolation process of extracellular vesicles. This disclosure thus relates to improving the accuracy of an extracellular vesicle-based diagnosis.

Abstract: The present disclosure provides methods and systems for processing nucleic acid molecules from one or more cells. A cell may be comprised in a cell bead. Processing may comprise uniquely identifying nucleic acid molecules. Nucleic acid molecules may be identified by barcoding. Barcoding may be combinatorial barcoding. Combinatorial barcoding may allow for generation of a large number of barcodes, thereby uniquely identifying nucleic acids from a large number of single cells. Combinatorial barcoding may be performed in successive operations. Successive operations may be performed in partitions.

Abstract: The present invention relates to the field of protein engineering, molecular imaging, and molecular diagnostics. More particularly, provided herein are novel synthetic aptamers produced using the Gyrl-like family of proteins as a scaffold. Methods of obtaining and using the synthetic mutein aptamers are also provided.

Abstract: The present invention is directed to a humanized CD19 single-chain variable fragment (scFv), comprising the amino acid sequence of SEQ ID NO: 8. The present invention is also directed to a CD19 chimeric antigen receptor fusion protein comprising from N-terminus to C-terminus: (i) a single-chain variable fragment (scFv) of the present invention, (ii) a transmembrane domain, (iii) at least one co-stimulatory domains, and (iv) an activating domain. The humanized anti-CD19 scFv of the present invention exhibits selective and high-affinity binding to CD19. CD19-CAR T cells based on humanized scFv of the present invention are useful to treat patients with B-cell malignancies including leukemia and lymphomas.

Abstract: The present invention provides a carboxylated nanodiamond-mediated CRISPR-Cas9 delivery system for gene editing comprising nanodiamond (ND) particles as the carriers of CRISPR-Cas9 components designed to introduce the mutation in a given gene for repairing a tissue damage.

Abstract: The present invention relates to recombinant Gram-negative bacterial strains and the use thereof for delivery of heterologous proteins into eukaryotic cells.

Abstract: Disclosed herein include systems, methods, compositions, and kits for sample identification. A sample indexing composition can comprise, for example, a protein binding reagent associated with a sample indexing oligonucleotide. Different sample indexing compositions can include sample indexing oligonucleotides with different sequences. Sample origin of cells can be identified based on the sequences of the sample indexing oligonucleotides. Sample indexing oligonucleotides can be barcoded using barcoded and lengthened using daisy-chaining primers.

Abstract: Reagents and methods for preparing nucleic acid samples for sequencing are provided. The reagents include multimeric barcoding reagents that comprise barcode regions linked together and a cell-binding moiety. The methods comprise contacting a nucleic acid sample comprising cells with a library of multimeric barcoding reagents, wherein each multimeric barcoding reagent comprises barcode regions linked together, and appending barcode sequences of a first multimeric barcoding reagent to sub-sequences of a target nucleic acid of a first cell, and appending barcode sequences of a second multimeric barcoding reagent to sub-sequences of a target nucleic acid of a second cell. Methods are also provided that comprise steps of internalising multimeric barcoding reagents into cells (e.g. by endocytosis) or exposing multimeric barcoding reagents to target nucleic acids by lysing cells or permeabilizing cell membranes.

Abstract: The present invention relates to the field of microfluidics and in particular to analysing the gene expression of a cell in response to a ligand expressed in the same microfluidic compartment. By barcoding the transcriptome of the cell and of the expression system generating the ligand, the effect of the ligand on the cell expression can be discerned. The invention provides microfluidic compartments and methods for this purpose.

Abstract: The present disclosure provides methods and compositions for the treatment of diseases and genetic disorders linked to SLC6A1 loss and/or misfunction. The methods and compositions of the present disclosure comprise rAAV vectors and rAAV viral vectors comprising transgene nucleic acid molecules comprising nucleic acid sequences encoding for a GAT1 polypeptide.

Abstract: Disclosed herein include systems, methods, compositions, and kits for the labeling of nucleic acids targets. In some embodiments, the methods comprise the use of target-specific primers and target-non-specific primers during library preparation (e.g., hybrid library preparation). In some embodiments, the methods increase the abundance of select low abundance targets during cDNA library preparation and/or sequencing library preparation.

Abstract: A method for obtaining a population of T cells from pluripotent stem cells, which includes a first step of obtaining hematopoietic stem cells and a second step of obtaining T cells. Also, the cell populations thus obtained according to this method and these cell populations for use as a medicament.

Abstract: The invention relates to a method for identifying and quantifying a polypeptide from a library of polypeptides. The method comprises the steps of: 1—providing a polypeptide library and a detection tag library, 2—generating a nested library comprising the polypeptides and the detection tags, 3—sequencing the nested library, 4—selecting a member of the nested library in one or several selection steps that are independent of a physical genotype-phenotype linkage, 5—isolating the detection tag from the selected polypeptide, 6—identifying and quantifying the detection tag by mass spectrometry, 7—obtaining the sequence of the selected polypeptide. The invention also relates to a collection of polypeptides, a collection of detection tags, and a collection of plasmid vectors.

Abstract: Chimeric antigen receptors (CARs) including an antigen binding domain specifically binding to coronavirus spike protein, nucleic acids encoding the CARs, vectors including nucleic acids encoding the CARs, and immune cells expressing the CARs are provided. Methods of treating a subject with coronavirus, including administering to the subject an immune cell expressing a disclosed CAR are also provided.

Abstract: Methods of uniquely labeling or barcoding molecules within a cell, a plurality of cells, and/or a tissue are provided. Kits for uniquely labeling or barcoding molecules within a cell, a plurality of cells, and/or a tissue are also provided. The molecules to be labeled may include, but are not limited to, RNAs, cDNAs, DNAs, proteins, peptides, and/or antigens.

Abstract: The present disclosure provides a high-throughput screening system and method for identification of novel drugs and drug targets. The method enables large-scale analysis of interactions between allogeneic pairs of target cells and immune cells by using an immune-bridge protein, library of guide RNA, and/or 3D tumor model.