Aspects of the present invention relate to the recombinant production of a mature complement system protein. Certain embodiments of the present invention relate to recombinant production of fully mature human complement Factor I protein (CFI). Included herein are details of an expression vector with which to recombinantly express fully mature human CFI from mammalian cells. Further disclosed are chromatography steps with which to purify recombinantly expressed CFI. Certain aspects of the present invention relate to the use of an expression system in gene therapy and the like. Certain embodiments of the present invention relate to use of said vector as a medicament, for example for use in the treatment of complement-mediated disorders.

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.

Micro-Organosphers, including Patient-Derived Micro-Organospheres (PMOSs), apparatuses and methods of making them, and apparatuses and methods of using them. Also described herein are methods and systems for screening a patient using these Patient-Derived Micro-Organospheres, including personalized therapies.

Disclosed is a method for culturing urine-derived kidney stem cells, which belongs to the field of cell biology. The method comprises the following steps: isolating cells from the urine, and then culturing the cells with a culture medium of urine-derived kidney stem cells on feeder cells to obtain the urine-derived kidney stem cells, wherein the feeder cells are fibroblasts, and the culture medium of urine-derived kidney stem cells contains 200-300 mL of DMEM medium, 200-300 mL of F12 medium, 20-70 mL of fetal bovine serum, 0.2-2 mM of L-glutamine, 1-14 ng/mL of insulin, 0.1-1 ng/mL of epidermal growth factor, 5-30 μg/mL of adenine, and 2-20 μg/mL of hydrocortisone. By using the method, kidney stem cells with high proliferation capacity and specificity can be obtained and applied, and thus the regenerative outcome of the kidney tissue after injury can be improved.

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.

The invention relates to a method for the formation of renal tubules by embedding individual renal cells into a synthetic hydrogel, which is based on polyethylene glycol as a component, and the culturing of the cells until tubule structures are formed. The culturing can be continued until the obtained tubule structures correspond in terms of size, structure, morphology and functionality to adult human renal tubules or are at least similar thereto.

A method for producing renal stromal cells, comprising a step (3) of culturing renal stromal precursors in a medium comprising a platelet derived growth factor receptor agonist to obtain renal stromal cells is provided as a technique for supplying renal stromal cells. This production method can further comprise a step (2) of inducing renal stromal precursors from neural crest cells, and a step (1) of culturing pluripotent stem cells in a medium comprising a GSK3β inhibitor, a TGFβ inhibitor, and retinoic acid and/or a derivative thereof to induce neural crest cells.

The present invention relates to microfluidic fluidic devices, methods and systems as microfluidic kidney on-chips, e.g. human Proximal Tubule-Kidney-Chip, Glomerulus (Kidney)-Chip, Collecting Duct (Kidney)-Chip. Devices, methods and systems are described for drug testing including drug transport and renal clearance. Further, such devices, methods and systems are used for determining drug-drug interactions and their effect upon renal transporter functions. Importantly, they may be used for pre-clinical and clinical drug development for treating kidney diseases and for personalized medicine.

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.

A method of reprogramming a differentiated renal cell towards a progenitor phenotype is disclosed. The method comprises up-regulating in the differentiated renal cell an expression of at least one pluripotency associated gene and/or at least one renal stem cell associated gene, thereby reprogramming the differentiated renal cell towards a progenitor phenotype. Cell populations generated thereby and uses thereof are also disclosed.