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.

Provided herein are methods for determining the presence of cancer (malignant versus benign), monitoring the progression of cancer, monitoring cancer relapse, monitoring the response to cancer therapy, or cancer staging in a subject, by evaluating CD33.sup.+/HLA-DR.sup.low, CD14.sup.+/HLA-DR.sup.low, CD66b.sup.+/HLA-DR.sup.low or, CD11b.sup.+/HLA-DR.sup.low MDSC for activation of a transcription factor. Transcription factors include, but are not limited to, STAT3, pSTAT3, HIF1α, or C/EBPβ. The MDSC phenotype can be CD33.sup.+HLA-DR.sup.lowHIF1α.sup.+/STAT3.sup.+, CD14.sup.+HLA-DR.sup.lowHIF1α.sup.+/STAT3.sup.+/pSTAT3.sup.+/C/EBPb.sup.+, CD66b.sup.+HLA-DR.sup.lowHIF1α.sup.+/STAT3.sup.+/pSTAT3.sup.+/C/EBPb.sup.+, CD33.sup.+HLA-DR.sup.lowHIF1α.sup.+/STAT3.sup.+/pSTAT3.sup.+/C/EBPb.sup.+, CD11b.sup.+HLA-DR.sup.lowHIF1α.sup.+/STAT3.sup.+/pSTAT3.sup.+/C/EBPb.sup.+, or CD11b.sup.+HLA-DR.sup.lowC/EBPβ.sup.+. Also provided herein are methods for inducing human MDSC from healthy donor peripheral blood mononuclear cells (PBMC) by co-culturing PBMC with human solid tumor cell lines and subsequently measuring their suppressive ability.

Methods are disclosed for identifying antibacterial compounds which inhibit propagation of selected spectrum bacteria, which bacteria use specific tRNA to code for Ala, Met, Ser, or Leu that other bacteria do not use. In one embodiment, the selected spectrum bacteria use GCA to code for Ala, whereas other bacteria use a different codon to code for alanine. The methods involve determining whether putative inhibitors promote or inhibit complex formation between the tRNA and a bacterial ribosome, or between the tRNA and an aminoacyl synthetase. Compounds which promote or inhibit complex formation can disrupt protein production, which bacteria need to propagate. The identified antibacterial compounds can selectively inhibit bacterial propagation. By limiting their effects to the selected spectrum bacteria, these compounds can treat or prevent specific bacterial infections without disrupting the normal bacterial flora, the patients' microbiome, or causing antibacterial resistance.

Provided is a cardiotoxicity assessment method which comprises: seeding cardiomyocytes in a culture container together with a culture medium; adding a drug to the culture medium in the culture container to bring the drug into contact with the cardiomyocytes; and measuring a heart disease biomarker secreted from the cardiomyocytes to assess cardiotoxicity of the drug, wherein at least a culture surface of the culture container is composed of an alicyclic structure-containing polymer, and the culture surface has a surface free energy of 30 to 37 mN/m.

Provided are assay systems for determining the therapeutic or toxic effect of a putative drug based on assaying its activity in cells which have been differentiated in vitro from stem cells, and induced to display a phenotype that resembles a disease to be treated.

Methods and compositions are provided for the treatment of a mitochondrial disease in an individual with the mitochondrial disease. Aspects of the methods include administering an inhibitor of a Pumilio-like protein and/or an inhibitor of a serine/arginine-rich family of pre-mRNA splicing factor (SR) protein to a subject. Also provided are methods, compositions, systems and kits for transdifferentiating target cells to neurons, which find use in producing neurons for the development of new therapies, for experimental evaluation, as a source of lineage- and cell-specific products, and the like, for example, for use in treating human disorders of the CNS.

Methods of treating kidney disease and protecting podocytes from injury are provided. Methods of screening agents for the treatment of kidney disease are also provided. In addition, methods of identifying structural or functional defects in a patient's podocytes and methods of identifying kidney disease causing agents in a patient's biological sample are also provided.

The presently disclosed subject matter relates to the generation of induced pluripotent stem cell (iPSC)-derived neuronal cell lines from subjects diagnosed with polyhydramnios-megalencephaly-symptomatic-epilepsy (PMSE) and assays making use of such cell lines to identify mammalian target of rapamycin (mTOR) signalling modulators as well as anti-epileptogenic compounds.

The present disclosure relates to a pharmaceutical composition for prevention or treatment of non-alcoholic steatohepatitis, the composition comprising, as an active ingredient, exosomes isolated from induced pluripotent stem cell-derived mesenchymal stem cells which have been or have not been treated with a pretreatment material. The exosomes of the present disclosure exhibit a more improved effect of preventing or treating non-alcoholic steatohepatitis, compared to those isolated from conventional mesenchymal stem cells and as such, can be advantageously used for relevant research and development, and productization.

The present disclosure provides compositions and methods for assaying the effectiveness of a potential therapeutic agent for treatment of an activated GαQ mutant cancer (e.g., melanoma or angiosarcoma) or an activated GαQ mutant melanocytic malignancy (e.g., Portwine stain or Sturge-Weber syndrome). Also disclosed herein are methods for treating an activated GαQ mutant cancer (e.g., melanoma or angiosarcoma) or an activated GαQ mutant melanocytic malignancy (e.g., Portwine stain or Sturge-Weber syndrome) in a subject in need thereof.