The present invention relates to a method of isolating exosomes. Specifically, the invention relates to a method comprising the steps of providing a sample including exosomes; identifying a cell-surface polypeptide on the exosomes; and isolating the exosomes using the cell-surface polypeptide on the exosomes. The exosomes isolated from by the methods of the invention can be studied for the purposes of biomarker identification, for the understanding of biological function and disease, and to find ways to target them with therapeutics.

Provided are methods for producing population of cells enriched for cells exhibiting sinoatrial node like characteristics. The cells can be produced from human pluripotent cells. Also provided are methods for using the SAN-like cells for identifying agents that can mitigate drug-induced cardiac toxicity. Also provided is a method for mitigating drug induced cardiotoxicity comprising administering to a subject an effective amount of physcion or a derivative thereof.

Provided are methods for producing population of cells enriched for cells exhibiting sinoatrial node like characteristics. The cells can be produced from human pluripotent cells. Also provided are methods for using the SAN-like cells for identifying agents that can mitigate drug-induced cardiac toxicity. Also provided is a method for mitigating drug induced cardiotoxicity comprising administering to a subject an effective amount of physcion or a derivative thereof.

The present application provides materials and methods for treating a patient with one or more conditions associated with DMPK whether ex vivo or in vivo. In addition, the present application provides materials and methods for editing and/or modulating the expression of DMPK gene in a cell by genome editing.

The present application provides materials and methods for treating a patient with one or more conditions associated with DMPK whether ex vivo or in vivo. In addition, the present application provides materials and methods for editing and/or modulating the expression of DMPK gene in a cell by genome editing.

The present invention relates to a gene therapy vector which is useful in the treatment or prevention of hypertrophic cardiomyopathy in a subject in need thereof. The gene therapy vector of the invention comprises a nucleic acid sequence encoding a cardiac sarcomeric protein and a cardiomyocyte-specific promoter which is operably linked to said nucleic acid sequence. The invention furthermore relates to a cell which comprises the gene therapy vector. Pharmaceutical compositions which comprise the gene therapy vector and/or a cell comprising said vector are also provided. In another aspect, the invention relates to a method for treating or preventing hypertrophic cardiomyopathy in a subject by introducing the gene therapy vector of the invention into a subject in need of treatment.

The present invention relates to a gene therapy vector which is useful in the treatment or prevention of hypertrophic cardiomyopathy in a subject in need thereof. The gene therapy vector of the invention comprises a nucleic acid sequence encoding a cardiac sarcomeric protein and a cardiomyocyte-specific promoter which is operably linked to said nucleic acid sequence. The invention furthermore relates to a cell which comprises the gene therapy vector. Pharmaceutical compositions which comprise the gene therapy vector and/or a cell comprising said vector are also provided. In another aspect, the invention relates to a method for treating or preventing hypertrophic cardiomyopathy in a subject by introducing the gene therapy vector of the invention into a subject in need of treatment.

The present invention provides extrcellular vesicles, such as exosomes, engineered to be loaded with miR-345, which may be further loaded with, e.g., miR-146a and let-7b, and/or further be depleted of miR-10a and/or miR-10b. The present invention also provides an assay method, wherein the amounts of miR-345, miR146a, and let-7b in a sample of extracellular vesicles are positively associated with potency, and wherein the amount of miR-10b in a sample of extracellular vesicles is negatively associated with potency.

The present invention provides extrcellular vesicles, such as exosomes, engineered to be loaded with miR-345, which may be further loaded with, e.g., miR-146a and let-7b, and/or further be depleted of miR-10a and/or miR-10b. The present invention also provides an assay method, wherein the amounts of miR-345, miR146a, and let-7b in a sample of extracellular vesicles are positively associated with potency, and wherein the amount of miR-10b in a sample of extracellular vesicles is negatively associated with potency.

Disclosed are subpopulations of mammalian stem cell- or mammalian progenitor cell-derived cardiomyocytes. The subpopulations of cardiomyocytes contain a portion of a population of mammalian stem cell- or mammalian progenitor cell-derived cardiomyocytes. The subpopulations of cardiomyocytes can be CD36.sup.+ subpopulations or CD36.sup.− subpopulations. Disclosed are methods of isolating and of using the subpopulations of cardiomyocytes, particularly in cardiac disease modeling, drug screening, cardiotoxicity testing, and cardiac regeneration/repair.