Abstract: This disclosure provides methods to modulate cardiac regeneration in a mammalian cardiac cell or progenitor, comprising contacting the mammalian cardiac cell with a DOT1L gene modulator.

Abstract: Provided herein are methods for treating a lysosomal transmembrane protein disease or disorder through ex vivo introduction of a nucleic acid molecule into hematopoietic stem and progenitor cells (HSPCs) followed by transplantation of the HSPCs into a subject in need of treatment. Also provided are vectors containing the nucleic acid molecule.

Abstract: Provided herein are methods for treating a lysosomal transmembrane protein disease or disorder through ex vivo introduction of a nucleic acid molecule into hematopoietic stem and progenitor cells (HSPCs) followed by transplantation of the HSPCs into a subject in need of treatment. Also provided are vectors containing the nucleic acid molecule.

Abstract: Provided herein are methods for treating a lysosomal transmembrane protein disease or disorder through ex vivo introduction of a nucleic acid molecule into hematopoietic stem and progenitor cells (HSPCs) followed by transplantation of the HSPCs into a subject in need of treatment. Also provided are vectors containing the nucleic acid molecule.

Abstract: The present invention relates to a small molecule high-throughput screening assay consisting of detectably labeled cardiac progenitor cells. The invention also describes a method of identifying small molecules from the high-throughput assay affecting cardiogenesis and/or modulating cardiac progenitor cell development. Also described are methods of stimulating maturation of cardiac progenitor cells using a GSK-3? inhibitor.

Abstract: The invention herein provides a mode of treating a disease associated with the regulation of T-Box and N-Myc gene, which includes cancers and heart disease in human and other subjects by identifying and administering a compound which modulates T-Box or N-Myc function. The invention also provides polynucleotides, polypeptides, vectors, cells, tissues and organisms useful in the identification and treatment of metabolic syndrome. A number of desirable cell proliferation and senescence regulating aspects are achieved by various embodiments of the present invention.

Abstract: The present invention provides isolated stem cells and progenitor cells, including HCN4-expressing CCS progenitor cells, HCN4- and Islet 1-expressing sinoatrial node cells or progenitor cells thereof, and Islet 1-expressing cardiovascular stem cells that do not express HCN4. The invention further provides differentiated cell types derived from the CCS progenitor cells and cardiovascular stem cells of the invention. In addition, the invention provides methods of differentiating the cells of the invention, methods for screening for agents that affect the differentiation of the cells of the invention, methods for evaluating the potential toxicity of drugs using the cells of the invention, cell therapy using the cells of the invention, and methods of treating heart arrhythmia by ablating HCN4-expressing cells.

Abstract: The present invention relates to a small molecule high-throughput screening assay consisting of detectably labeled cardiac progenitor cells. The invention also describes a method of identifying small molecules from the high-throughput assay affecting cardiogenesis and/or modulating cardiac progenitor cell development. Also described are methods of stimulating maturation of cardiac progenitor cells using a GSK-3? inhibitor.

Abstract: The invention herein provides a mode of treating a disease associated with the regulation of T-Box and N-Myc gene, which includes cancers and heart disease in human and other subjects by identifying and administering a compound which modulates T-Box or N-Myc function. The invention also provides polynucleotides, polypeptides, vectors, cells, tissues and organisms useful in the identification and treatment of metabolic syndrome. A number of desirable cell proliferation and senescence regulating aspects are achieved by various embodiments of the present invention.

Abstract: The invention herein provides a mode of treating a disease associated with the regulation of T-Box and N-Myc gene which includes cancers and heart disease in human and other subjects by identifying and administering a compound which modulates T-Box or N-Myc function. The invention also provides polynucleotides, polypeptides, vectors, cells, tissues and organisms useful in the identification and treatment of metabolic syndrome. A number of desirable cell proliferation and senescence regulating aspects are achieved by various embodiments of the present invention.

Abstract: The present invention relates to a small molecule high-throughput screening assay consisting of detectably labeled cardiac progenitor cells. The invention also describes a method of identifying small molecules from the high-throughput assay affecting cardiogenesis and/or modulating cardiac progenitor cell development. Also described are methods of stimulating maturation of cardiac progenitor cells using a GSK-3? inhibitor.

Abstract: The invention herein provides a mode of treating a disease associated with the regulation of T-Box and N-Myc gene which includes cancers and heart disease in human and other subjects by identifying and administering a compound which modulates T-Box or N-Myc function. The invention also provides polynucleotides, polypeptides, vectors, cells, tissues and organisms useful in the identification and treatment of metabolic syndrome. A number of desirable cell proliferation and senescence regulating aspects are achieved by various embodiments of the present invention.

Abstract: The present invention provides in vitro methods of expansion and propagation of undifferentiated progenitor cells and more specifically undifferentiated progenitor cells containing Islet1, a marker apparently unique to proliferating cardiac stem cells. Methods are described for isolation of stem cell populations as well as for provoking expansion and propagation of undifferentiated progenitor cells without differentiation, to provide cardiac repair or improve cardiac function, for example.

Abstract: The present invention relates to a human type-5 recombinant adenovirus vector for achieving cardiac restricted transcription involving utilization of the cardiomyocyte-restricted cardiac ankyrin repeat protein (CARP) promoter with inclusion of the inverted terminal repeat sequences from human adeno-associated virus (AAV). Using green fluorescent protein (GFP) as a marker gene, the recombinant adenovirus vector (Ad/CG/ITR) is shown to direct transgene expression to myocardial tissue in vivo and in vitro in mouse models.