Abstract: Products and methods for treating dystroglycanopathies and laminin-deficient muscular dystrophies are provided. In the methods, a protein including a linker domain, such as the heparin-binding domain of Heparin-Binding Epidermal Growth Factor-Like Growth Factor (HBEGF), is delivered to patients.

Abstract: The present invention relates to a polypeptide comprising a piggyBac transposase or a fragment or a derivative thereof having transposase function comprising at least one amino acid substitution. Further, the present invention relates to a transposable element comprising a piggyBac or piggyBac-like left repeat sequence and left internal repeat sequence, wherein the left internal repeat sequence comprises at least one nucleotide modification. Furthermore, the present invention relates to a kit comprising the above transposase and/or transposable element. In addition, the present invention relates to a targeting system comprising the above transposase and/or transposable element.

Abstract: An in vitro method of preparing insulin-producing cell clusters for transplantation into a subject, comprising (a) seeding pancreatic progenitor cells onto a three-dimensional, porous scaffold at a seeding density greater than about 12.5 million cells per cm3 of scaffold and less than about 150 million cells per cm3 of scaffold, wherein the scaffold comprises a plurality of pores having an average pore diameter greater than about 225 ?m and less than about 550 pm, and (b) culturing the cells on the scaffold for more than about 3 days in culture medium to obtain insulin-producing cell clusters within the pores of the scaffold for transplantation into a subject. In exemplary aspects, the pancreatic progenitor cells are cells derived from pluripotent stem cells.

Abstract: In an aspect, disclosed herein are physiological devices and systems, and components thereof, used to evaluate cardiac parameters and arrhythmogenic mechanisms. This abstract is intended as a scanning tool for purposes of searching in the particular art and is not intended to be limiting of the present invention.

Abstract: The present invention provides differentiated neural cells and methods for making differentiated neural cells from pluripotent stem cells (PSC) at an industrial scale sufficient for high-throughput assays. The methods of the invention allow billions of PSCs and/or neural cells differentiated from the PSCs to be cryopreserved and expanded at multiple steps.

Abstract: Compositions and methods for repairing or regenerating damaged tissue are disclosed. In particular, the invention relates to methods of delivering secreted factors from stem cells to tissues in order to immobilize and concentrate such secreted factors at or under the surface of damaged tissue to promote tissue regeneration.

Abstract: The present invention provides a production method for a three-dimensional muscle tissue including the steps of: preparing an approximately rectangular first cell module containing skeletal myoblasts in a hydrogel, and having a plurality of approximately rectangular holes parallel to each other, and an approximately rectangular second cell module containing skeletal myoblasts in a hydrogel, and having a plurality of approximately rectangular holes parallel to each other at positions different from those of the first cell module in a vertical direction; alternately stacking the prepared first cell module and the prepared second cell module to obtain a stack; subjecting the skeletal myoblasts contained in the obtained stack to proliferation culture; and inducing the proliferated skeletal myoblasts to differentiate into myotubes.

Abstract: The invention relates to a method for generating a cardiac tissue mimetic, comprising the steps of mixing cardiomyocytes (CM) and fibroblasts (FB) at a ratio from 2.5:1 to 10:1, thereby providing a first mixture, incubating said first mixture, such that said cardiomyocytes and said fibroblasts form a spherical structure, adding endothelial cells (EC) to said spherical structure at a ratio of cardiomyocytes (CM) to endothelial cells (EC) from 1.5:1 to 4:1, thereby providing a second mixture, and incubating said second mixture, such that a cardiac tissue mimetic is formed. The invention further relates to a cardiac tissue mimetic comprising cardiomyocytes (CM), endothelial cells (EC), and fibroblasts (FB), wherein said cardiac tissue mimetic comprises sarcomeric structures and vascular structures. Further aspects of the invention are the cardiac tissue mimetic for use in a method for heart tissue repair or replacement and a method for screening a compound using a cardiac tissue mimetic.

Abstract: Provided herein are methods of using mesenchymal stromal/stem cell (MCS) exosomes in the treatment of diseases associated with thymic dysfunction. In some embodiments, the MSC exosomes restore thymic architecture and development in a subject having thymic dysfunction (e.g., caused by exposure to hyperoxia). In some embodiments, the subject is a human subject (e.g., a human neonate).

Abstract: A method for producing enteric neural precursors, comprising the steps of: (1) providing enteric neural precursors; and (2) culturing the enteric neural precursors in a medium comprising an ERBB3 agonist and/or an ERBB4 agonist is provided as a technique for allowing enteric neural precursors to proliferate by culture while maintaining their differentiation capacity into enteric nerve cells and glial cells.

Abstract: Methods and compositions for introducing an exogenous mitochondrion harvested from a donor cell into a recipient cell are provided. Also provided are methods and compositions for producing a modified mitochondrion and treating mitochondrial disease in an individual with the mitochondrial disease. Further provided are modified mitochondria, genetically modified mitochondria, genetically modified cells, isolated compositions and pharmaceutical compositions for practicing the subject methods.

Abstract: Disclosed are mesenchymal stem cell growth factor compositions and methods of their use to treat skin disorder, alleviate the effects of aging, treat wounds, orthopedic disorders, sexual dysfunction and/or reduce inflammation.

Abstract: Compositions and methods are provided for generation of a population of monohormonal from human pluripotent cells. The method comprises sequentially exposing pluripotent stem cells to medium comprising CHIR99021 and Activin A; medium comprising Activin A; medium comprising FGF7; medium comprising retinoic acid, LDN193189, SANT1; medium comprising retinoic acid, LDN193189, SANT1, EGF and FGF2; and differentiation medium. A majority of cells in the population are monohormonal cells produce insulin, but not glucagon, somatostatin or ghrelin.

Abstract: This presently disclosed subject matter relates to an in vitro cell culture comprising a cell monolayer comprising mucus producing cells and a mucus layer, and methods of making and using the same. The methods including culturing mucus producing cells on a cell support structure under conditions to establish a mucus layer on the luminal side of the cell monolayer, thereby producing a live cell construct comprising a cell monolayer comprising mucus producing cells and a mucus layer. The mucus layer can be substantially impenetrable to micro-objects, and have a thickness of about 1 micron to about 1 cm.

Abstract: The invention relates to a minimal U6 pol III promoter. The invention also concerns a nucleic acid construct comprising the minimal U6 pol III promoter, a vector comprising the minimal U6 pol III promoter, methods involving the minimal U6 pol III promoter, and uses for the minimal U6 pol III promoter.

Abstract: The present application is directed to methods and compositions that are useful for reducing the number of myeloid-derived suppressor cells (MDSC), tumor associated macrophages (TAM), or both in a subject. The methods include administering an antigen binding protein that binds to an antigen expressed by MDSC and/or TAM, or administering a modified T cell or NK-92 cell that expresses an antigen binding protein that binds to an antigen expressed by MDSC and/or TAM, or a combination of both, to a subject. For example, the antigen binding protein can bind to CD33 expressed by MDSC and/or TAM. The methods and compositions are useful for treating a disease associated with MDSC and/or TAM infiltration into a tissue or tumor.

Abstract: A method for inducing reprogramming of neural stem cells from urine cells by introducing mRNAs of reprogramming factors Oct4, Sox2, Klf4, and Glis1 is disclosed. A composition for the prevention or treatment of neurological damage diseases with the neural stem cells induced by the method as an active ingredient is disclosed.

Abstract: Provided herein are methods of stimulating or increasing the proliferation of a Treg cell, compositions including a population of Treg cells generated by these methods, and methods of treating a subject using these compositions.

Abstract: A method for culturing colorectal cancer solid tumor primary cells and colorectal cancer ascites primary tumor cells and supporting reagents. A method for culturing colorectal cancer solid tumor primary cells and colorectal cancer ascites primary tumor cells and supporting reagents. Colorectal cancer solid tumor tissues are treated with mild cell dissociation reagents, and colorectal cancer cells are isolated from ascites with a mild method, thereby ensuring the vitality of cancer cells to the greatest extent. A special serum-free medium is prepared, and colorectal cancer solid tumor-derived tumor cells are cultured in vitro with a suspension culture system to ensure normal expansion of the cancer cells while eliminating the interference of normal cells to the greatest extent. The colorectal cancer primary cell culture obtained by the method are usable for in vitro experiments, second-generation sequencing, building of animal models, and building of cell lines at multiple cell levels.

Abstract: The present disclosure provides ex vivo chamber-specific cardiac tissues, methods for generating the cardiac tissues in a bioreactor, and methods of using the cardiac tissues. Examples of cardiac tissues that can be generated include, but are not limited to, atrial tissues, ventricular tissues, and composite tissues having an atrial tissue connected to a ventricular tissue.