The present invention relates to a polynucleotide comprising at least one promoter, at least one expressible construct, and an S/MAR element, wherein said polynucleotide is an integration construct or a non-integrative vector construct, wherein said S/MAR element is located downstream of said promoter and of said expressible construct, and wherein said S/MAR element is flanked by a splice donor and a splice acceptor. The present invention also relates to a composition and a host cell comprising said polynucleotide, as well as to uses and methods related thereto.

The present disclosure provides an in vitro mammalian cell that is genetically modified to provide for enhanced production of adeno-associated virus (AAV) virions. The mammalian cells can be used to produce AAV virions, e.g., recombinant AAV virions that include a heterologous nucleic acid encoding a gene product; the present disclosure thus provides methods for producing an AAV virion, which may be a recombinant AAV virion.

Provided herein are, inter alia, extracellular products (e.g., vesicles such as microvesicles, e.g., exosomes) produced by renal cells (such as bioactive renal cells, e.g., selected renal cells). Methods of altering components (such as miRNAs or proteins) of vesicles produced by cells, as well as methods of producing vesicles comprising various compounds are also included. Also provided are diagnostic and treatment methods

A method is provided for simultaneously producing both nephron progenitor cells and ureteric epithelial progenitor cells including the step of contacting intermediate mesoderm cells with: fibroblast growth factor 9 and/or fibroblast growth factor 20 and optionally, one or more selected from the group consisting of: bone morphogenic protein 7; heparin; a Wnt agonist; retinoic acid; and an RA antagonist. The concentrations of Wnt agonist, retinoic acid and/or RA antagonist may be manipulated to favour the relative production of nephron progenitor cells and ureteric epithelial progenitor cells. The intermediate mesoderm cells are ultimately derived from human pluripotent stem cells via a posterior primitive streak stage. The nephron progenitor cells and ureteric epithelial progenitor cells may have end uses such as for kidney repair and regeneration, bioprinting of kidneys and screening compounds for nephrotoxicity.

Disclosed is a method for inducing direct reprogramming of urine cells into renal progenitor cells and a pharmaceutical composition including the renal progenitor cells reprogrammed by the method for preventing or treating renal cell injury disease. The method can make the mass production of customized reprogrammed renal progenitor cells by using urine cells, which are somatic cells easily and repeatedly obtainable without inconvenience and pain and as such, can be applied to incurable disease fields expandable to the renal injury therapy and kidney regeneration fields and to the production of cell therapy products.

The present invention provides: a method for producing renal progenitor cells from intermediate mesoderm cells, which comprises a step of culturing intermediate mesoderm cells in a medium containing a TGFβ signaling activator(s) and a BMP inhibitor(s); the renal progenitor cells produced by the method; a pharmaceutical composition comprising the renal progenitor cells; and a therapeutic drug for kidney diseases comprising the renal progenitor cells.

Tunneling nanotube (TNT) cells, comprising a TNT promoting factor (TPF); and a biomolecule cargo overexpressed by the TNT cell, and methods of use thereof for delivery of the biomolecule cargo from TNT cells to neighboring cells.

A method for acquiring and producing high-purity renal progenitor cells from a renal progenitor cell population into which pluripotent stem cells are induced to differentiate, by identifying a cell surface antigen marker specific to renal progenitor cells. The high-purity renal progenitor cells can be used in regenerative medicine for renal diseases, such as renal failure.

The disclosure describes novel systems, methods, and compositions for the manipulation of nucleic acids in a targeted fashion. The disclosure describes non-naturally occurring, engineered CRISPR systems, components, and methods for targeted modification of nucleic acids. Each system includes one or more protein components and one or more nucleic acid components that together target nucleic acids.

An object of the present invention is to provide a method for selecting cells, an apparatus for selecting cells, and an apparatus system for selecting cells, which enables evaluation of a damage resistance of the cells in a suspension culture system. According to the present invention, provided is a method for selecting cells including introducing cells into a micro flow passage and allowing the cells to pass through the micro flow passage, evaluating a damage resistance of the cells to damage received by the cells due to passing through the micro flow passage, and selecting the cells based on the evaluation of the damage resistance.