Abstract: The present invention provides genetic markers for identifying engraftable human cardiac ventricular progenitor cells. The engraftment markers of the invention include angiogenic markers and extracellular matrix markers. Human ventricular progenitor cells expressing these markers are capable of forming ventricular tissue in vivo that is vascularized and supported by an extracellular matrix. Methods of engrafting human cardiac ventricular progenitor cells by transplanting into a subject progenitor cells that express the engraftment markers are also provided.

Abstract: This document provides methods and materials related to vesicular stomatitis viruses. For example, replication-competent vesicular stomatitis viruses, nucleic acid molecules encoding replication-competent vesicular stomatitis viruses, methods for making replication-competent vesicular stomatitis viruses, and methods for using replication-competent vesicular stomatitis viruses to treat cancer or infectious diseases are provided.

Abstract: Provided herein are recombinant expression vector systems for expression by stem cells that allow for selective elimination of stem cell-derived tumorigenic cells, while preserving stem cell-derived therapeutic cells. The systems provided herein provide a means by which modified stem cells can be safely transplanted into subjects. In exemplary embodiments, the system comprises at least two suicide genes, one of which is specifically inactivated in cells exhibiting the desired phenotype and one of which is activated when the cell comprising the recombinant expression vector system behaves similarly to or the same as a tumor cell.

Abstract: Provided herein are methods of treatment of individuals having an immune-related disease, disorder or condition, for example, inflammatory bowel disease, graft-versus-host disease, multiple sclerosis, rheumatoid arthritis, psoriasis, lupus erythematosus, diabetes, mycosis fungoides (Alibert-Bazin syndrome), or scleroderma using placental stem cells or umbilical cord stem cells.

Abstract: The invention provides a method for producing a ciliary marginal zone stem cell induced to differentiate from a pluripotent stem cell, including either the following step (1) or step (2), or both of these steps: (1) a step of floating culturing cells obtained from a cell aggregate containing a ciliary marginal zone-like structure induced to differentiate from pluripotent stem cells, thereby obtaining a retinosphere; and (2) a step of collecting stage specific embryonic antigen-1 positive cells from cells obtained from a cell aggregate containing a ciliary marginal zone-like structure induced to differentiate from pluripotent stem cells.

Abstract: The present invention relates to transgenic blue ornamental fish, as well as methods of making such fish by in vitro fertilization techniques. Also disclosed are methods of establishing a population of such transgenic fish and methods of providing them to the ornamental fish industry for the purpose of marketing.

Abstract: Cell penetrating peptides that target many cells types including cells of the retina and cornea with high efficiency are provided herein. These peptides can be used to deliver cargo molecules across a plasma membrane, without the need for chemical conjugation. Compositions and viral vectors comprising these cell-penetrating peptides are also provided. Methods of using the peptides, compositions and viruses to deliver various agents to target cells and tissues are also provided.

Abstract: An object of the present invention is to provide, for example, a more practical wound healing accelerator that more effectively accelerates wound healing. More specifically, a feature of the present invention is to provide, for example, a more practical wound healing accelerator that is easily obtained in a larger amount than that of peripheral blood platelet and has a better wound healing effect than that of peripheral blood platelet. The present invention employs a platelet-like cell population coexpressing one or more platelet surface markers and one or more mesenchymal cell surface markers. A wound healing accelerator containing the platelet-like cell population is a more practical wound healing accelerator that more effectively accelerates wound healing. The platelet-like cell population is easily obtained in a larger amount than that of peripheral blood platelet and has a better wound healing effect than that of peripheral blood platelet.

Abstract: Compositions of genetically modified beta-like cells are encompassed. Also encompassed are methods of treatment of type 1 diabetes using these compositions or compositions that inhibit the function of the identified genes.

Abstract: The present invention is in the field of CRISPR-Cas system for genome targeting. The present invention relates to new engineered Cas9 scaffolds and uses thereof. More particularly, the present invention relates to methods for genome targeting, cell engineering and therapeutic application. The present invention also relates to vectors, compositions and kits in which the new Cas9 scaffolds of the present invention are used.

Abstract: The present invention relates to compositions and methods comprising cell surface markers for pluripotent-derived cells, in particular, pancreatic endoderm-type cells, derived from pluripotent stem cells.

Abstract: The present disclosure provides recombinant adeno-associated virus virions with variant capsid protein, where the recombinant AAV (rAAV) virions exhibit one or more of increased ability to cross neuronal cell barriers, increased infectivity of a neural stem cell, increased infectivity of a neuronal cell, and reduced susceptibility to antibody neutralization, compared to a control AAV, and where the rAAV virions comprise a heterologous nucleic acid. The present disclosure provides methods of delivering a gene product to a neural stem cell or a neuronal cell in an individual. The present disclosure also provides methods of modifying a target nucleic acid present in a neural stem cell or neuronal cell.

Abstract: An example genetically engineered effector cell comprises an exogenous polynucleotide sequence that includes a receptor element, an actuator element, and an effector element. The receptor element encodes a chimeric antigen receptor (CAR) comprising an extracellular antigen binding domain operably linked to a transmembrane domain, and an intracellular signaling domain, wherein the extracellular antigen binding domain recognizes an antigen on a surface of a Severe Acute Respiratory Syndrome Coronavirus 2 (SARS-CoV-2)-infected cell. The actuator element encodes a transcription factor binding site that upregulates synthesis of an antiviral protein. The effector element encodes the antiviral protein operably linked to a signal peptide, wherein, in response to the antigen binding domain of the CAR binding to the antigen of SARS-CoV-2-infected cell, the engineered effector cell is configured to activate and, to synthesize and secrete the antiviral protein.

Abstract: The present invention describes mRNA usage improving and/or translation-enhancing nucleic acid sequences, nucleic acid constructs comprising such sequences, and host cells comprising such nucleic acid constructs. The invention further pertains to a method for expressing a protein of interest in a cell or organism using such nucleic acid sequences, as well as their uses for increasing intergration of such nucleic acid construct into a genome, for enhancing mRNA usage and/or translation of a recombinantly expressed polypeptide, and for increasing the number of transformants upon transformation of a cell with such nucleic acid construct.

Abstract: The disclosure features compositions and methods for the treatment of sensorineural hearing loss and auditory neuropathy, particularly forms of the disease that are associated with mutations in otoferlin (OTOF), by way of OTOF gene therapy. The disclosure provides a variety of compositions that include a first nucleic acid vector that contains a polynucleotide encoding an N-terminal portion of an OTOF protein and a second nucleic acid vector that contains a polynucleotide encoding a C-terminal portion of an OTOF protein. These vectors can be used to increase the expression of OTOF in a subject, such as a human subject suffering from sensorineural hearing loss.

Abstract: The present invention relates to compositions comprising a decellularized tissue. The present invention also provides an engineered three dimensional lung tissue exhibiting characteristics of a natural lung tissue. The engineered tissue is useful for the study of lung developmental biology and pathology as well as drug discovery.

Abstract: The present invention relates to compositions and methods for enhancing T cell metabolism and activity for more effective adoptive T cell therapy. By expressing an intracellular signaling molecule in T cells, the T cells are metabolically enhanced with improved cytotoxicity and resistance to immunosuppression imposed by tumor microenvironments. One aspect includes a modified T cell and pharmaceutical compositions comprising the modified cells for adoptive cell therapy and treating a disease or condition associated with enhanced immunity.

Abstract: Provided herein are CRISPR/Cas9 complexes and method of using same.

Abstract: The present disclosure relates to nucleic acid promoter sequences that are able to specifically express genes operatively linked to the promoter in brainstem and spinal motor neuron cells, and to methods for using such promoters to selectively express genes in motor neurons in vitro and in vivo. It is based, at least in part, on the discovery that the nucleic acid of SEQ ID NO: 1 functioned as a motor neuron-specific promoter and was successful in expressing transgenes in motor neuron cells in vivo. The present disclosure also relates to compositions that can increase the activity or expression level of miR-218 and to compositions that can decrease the expression of miR-218 target nucleic acids.

Abstract: Provided herein are engineered TSC2 polypeptides, and nucleic acid sequences encoding them, in which the ability of a serine residue to be phosphorylated is altered. In some aspects, the TSC2 serine residue cannot be phosphorylated (e.g., by substituting the serine residue with an alanine residue). In some aspects, the TSC2 serine acts as if it is constitutively phosphorylated (e.g., by substituting the serine residue with a glutamic acid residue). Also provided herein are engineered immune cells comprising altered TSC2 polypeptides or nucleic acid sequences encoding them, and methods of making and using such engineered immune cells.