The present disclosure, in some aspects, is directed to methods for screening and identifying compounds that modulate one or more characteristics associated with a condensate comprising a RBM20 polypeptide and/or the RBM20 polypeptide. In other aspects, the disclosure is directed to systems and composition components thereof, such as cellular models useful for the methods described herein.

The present disclosure relates to an inhibitor of Dynamin 2 for use in the treatment of centronuclear myopathies. The present disclosure relates to pharmaceutical compositions containing Dynamin 2 inhibitor and to their use for the treatment of centronuclear myopathies. It also deals with a method for identifying or screening molecules useful in the treatment of a centronuclear myopathy.

A well for electrically stimulating at least one cell. The well includes a bottom portion and comprises an adaptive electrode arrangement for introducing an electric field into the well. The adaptive electrode arrangement includes a pair of electrodes disposed within the well. Each electrode of the pair of electrodes has a distal end and is independently and axially displaceable relative to the other electrode and the bottom portion of the well. The distal end of each electrode of the pair of electrodes is in contact with the bottom portion of the well, ensuring a uniform and constant electric field is applied within the well.

Methods and compositions for the production of cardiomyocytes and cardiac organoids from the differentiation of pluripotent stem cells in three-dimensional (3D) culture are provided. Rock inhibitor, which has been ubiquitously used as a medium supplement to prevent apoptosis during handling and culture of pluripotent stem cells, compromises the capacity of cardiac differentiation of pluripotent stem cells in 3D culture at the most commonly used concentrations.

The electrical pacemakers currently being used for the therapeutic approaches for treatment of “sick sinus syndrome” are not hormonally regulatable and entail risks through infections or premature battery discharge. These problems could be overcome by means of “biological cardiac pacemakers” obtained from pluripotent stem cells (PSCs). It has been shown that the controlled differentiation of stem cells with TBX, inductors of sinoatrial node cells, and an additional Myh6 promoter-specific antibiotic selection can give cardiomyocyte aggregates consisting to an extent of more than 80% of physiologically functional pacemaker cells. These induced sinoatrial bodies (“iSABs”) for the first time exhibited very high beat frequencies (300-400 bpm), similar to those in a murine heart, and were able to stably rhythmically stimulate heart muscle cells ex vivo. In the iSAB transcriptome decoded by means of RNA-seq, it was possible to assign almost all the genes to the ontologies of heart function/heart development and the structures of contractile cells. Overall, this is the first example of a high-purity functional sinoatrial tissue derived from stem cells, which means that a crucial step for future cell therapy and the testing of medicaments in vitro is being implemented.

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.

A use of an MYOG gene as a target in the preparation of a drug for treating a cardiomyocyte apoptosis-associated cardiovascular disease (CVD) is provided. By constructing angiotensin II-induced human induced pluripotent stem cell-differentiated cardiomyocyte apoptosis models in vitro, the present disclosure reveals for the first time the role of the transcription factor MYOG in the inhibition of cardiomyocyte apoptosis and provides a theoretical and scientific basis for drug research and development of cardiomyocyte apoptosis-associated CVDs. Cardiomyocyte apoptosis model becomes a new target for CVD drug research and development.

Embodiments described herein relate generally to a three-dimensional ex vivo skeletal muscle tissue comprising a hydrogel and a plurality of cells that includes skeletal muscle cells, at least a portion of the cells being encapsulated inside the hydrogel. In some embodiments, the skeletal muscle tissue is characterized by one or more contractions in response to an electrical and/or chemical stimulation.

A method of manufacturing a microstructure comprises printing a positive mold structure, filling the positive mold structure with a second material to form an elastically deformable negative mold structure, filling the negative mold structure with a third material to form the microstructure, and releasing the microstructure from the negative mold structure. Advantageously, the negative mold structure can be stretched to facilitate the release of the microstructure. For example, the microstructure comprises a chamber with capped micropillars for the generation and/or analysis of muscle tissue.

Provided herein are tissues containing semiconductor nanomaterials and methods of preparing and using the same.