Abstract: Some embodiments provide a method of treating skeletal muscular myopathy, e.g., Duchenne muscular dystrophy (DMD), with cardiosphere-derived cells (CDCs), wherein a therapeutically effective amount of CDCs is delivered to a targeted dystrophic skeletal muscle. Some embodiment enable delivery of a therapeutically effective amount of CDCs via intramuscular injection directly at a skeletal muscle or systemic administration, e.g., intravenous injection, in a single dose or multiple doses, to treat a targeted dystrophic skeletal muscle. Some embodiments provide a method for improving exercise capabilities in DMD patients. Additional embodiments relate to exosome mediated transfer of noncoding RNAs ameliorates Duchenne muscular dystrophy by restoring dystrophin in heart and skeletal muscle. Delivery of noncoding RNA species found in CDC-derived exosomes mimics the ability of CDCs and CDC-derived exosomes to increase dystrophin protein levels.

Abstract: The present invention relates to a method of treating a chemical injury of the eye, in particular alkali burn of the cornea, as well as ocular GVHD and similar inflammatory ocular conditions, with extracellular vesicles, in particular exosomes obtained from human cardiospheres or cardiosphere-derived cells. The present invention also provides a formulation comprising extracellular vesicles, in particular exosomes obtained from human cardiospheres or cardiosphere-derived cells, for subconjunctival or topical administration to the eye in the treatment of a chemical injury of the eye, in particular alkali burn of the cornea, as well as ocular GVHD and similar inflammatory ocular conditions.

Abstract: The present invention relates to a method of treating acute or chronic systemic graft-versus-host disease (GVHD) with extracellular vesicles, e.g., exosomes obtained from human cardiospheres or cardiosphere-derived cells (CDCs), wherein systemic GVHD involves, e.g., at least two organs selected from the group consisting of the skin, mucosa, gastrointestinal tract, liver, lungs, joints and fascia, genitalia, and eyes. The present invention also provides a pharmaceutical formulation comprising extracellular vesicles, e.g., exosomes obtained from human cardiospheres or CDCs, for systemic administration, e.g., intravenous infusion, to a human subject in need of treatment of systemic GVHD.

Abstract: Some embodiments provide a method of treating skeletal muscular myopathy, e.g., Duchenne muscular dystrophy (DMD), with cardiosphere-derived cells (CDCs), wherein a therapeutically effective amount of CDCs is delivered to a targeted dystrophic skeletal muscle. Some embodiment enable delivery of a therapeutically effective amount of CDCs via intramuscular injection directly at a skeletal muscle or systemic administration, intravenous injection, in a single dose or multiple doses, to treat a targeted dystrophic skeletal muscle. Some embodiments provide a method for improving exercise capabilities in DMD patients. Additional embodiments relate to exosome, mediated transfer of noncoding RNAs ameliorates Duchenne muscular dystrophy by restoring dystrophin in heart and skeletal muscle. Delivery of noncoding RNA species found in CDC-derived exosomes mimics the ability of CDCs and CDC-derived exosomes to increase dystrophin protein levels.

Abstract: 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.

Abstract: The present invention relates to a method of treating acute or chronic systemic graft-versus-host disease (GVHD) with extracellular vesicles, e.g., exosomes obtained from human cardiospheres or cardiosphere-derived cells (CDCs), wherein systemic GVHD involves, e.g., at least two organs selected from the group consisting of the skin, mucosa, gastrointestinal tract, liver, lungs, joints and fascia, genitalia, and eyes. The present invention also provides a pharmaceutical formulation comprising extracellular vesicles, e.g., exosomes obtained from human cardiospheres or CDCs, for systemic administration, e.g., intravenous infusion, to a human subject in need of treatment of systemic GVHD.

Abstract: Several embodiments relate to methods of generating cells with therapeutic potency. Several embodiments relate to generating cells as a source of exosomes with therapeutic potency. The cells and exosomes with therapeutic potency are useful for repairing and/or regenerating damaged or diseased tissue, for example.

Abstract: The present invention provides a method of inducing activation of a non-potent or insufficiently potent cell to convert the cell into a tissue-effector cell, thereby producing an activation-induced tissue-effector cell suitable for use in cell therapy—e.g., an activated specialized tissue-effector cell (ASTEC) suitable for cell therapy for a particular tissue type. The present invention further provides activation-induced tissue-effector cells produced thereby, as well as extracellular vesicles, e.g., exosomes, derived therefrom (e.g., ASTEX). The present invention further provides a method of improving the efficacy of a cell therapy by converting non-potent or insufficiently potent cells into activation-induced tissue-effector cells having increased potency suitable for cell therapy.

Abstract: The present invention relates to a method of treating a chemical injury of the eye, in particular alkali burn of the cornea, as well as ocular GVHD and similar inflammatory ocular conditions, with extracellular vesicles, in particular exosomes obtained from human cardiospheres or cardiosphere-derived cells. The present invention also provides a formulation comprising extracellular vesicles, in particular exosomes obtained from human cardiospheres or cardiosphere-derived cells, for subconjunctival or topical administration to the eye in the treatment of a chemical injury of the eye, in particular alkali burn of the cornea, as well as ocular GVHD and similar inflammatory ocular conditions.

Abstract: The present invention relates to a method of treating dermatitis, in particular radiation-induced dermatitis, with extracellular vesicles, in particular exosomes obtained from human cardiospheres or cardiosphere-derived cells. The present invention also provides a formulation comprising extracellular vesicles, in particular exosomes obtained from human cardiospheres or cardiosphere-derived cells, for use in the treatment of dermatitis, in particular radiation-induced dermatitis.

Abstract: Some embodiments provide a method of treating skeletal muscular myopathy, e.g., Duchenne muscular dystrophy (DMD), with cardiosphere-derived cells (CDCs), wherein a therapeutically effective amount of CDCs is delivered to a targeted dystrophic skeletal muscle. Some embodiment enable delivery of a therapeutically effective amount of CDCs via intramuscular injection directly at a skeletal muscle or systemic administration, intravenous injection, in a single dose or multiple doses, to treat a targeted dystrophic skeletal muscle. Some embodiments provide a method for improving exercise capabilities in DMD patients. Additional embodiments relate to exosome, mediated transfer of noncoding RNAs ameliorates Duchenne muscular dystrophy by restoring dystrophin in heart and skeletal muscle. Delivery of noncoding RNA species found in CDC-derived exosomes mimics the ability of CDCs and CDC-derived exosomes to increase dystrophin protein levels.

Abstract: The present invention relates to a method of treating acute or chronic systemic graft-versus-host disease (GVHD) with extracellular vesicles, e.g., exosomes obtained from human cardiospheres or cardiosphere-derived cells (CDCs), wherein systemic GVHD involves, e.g., at least two organs selected from the group consisting of the skin, mucosa, gastrointestinal tract, liver, lungs, joints and fascia, genitalia, and eyes. The present invention also provides a pharmaceutical formulation comprising extracellular vesicles, e.g., exosomes obtained from human cardiospheres or CDCs, for systemic administration, e.g., intravenous infusion, to a human subject in need of treatment of systemic GVHD.

Abstract: The present invention relates to a method of treating dermatitis, in particular radiation-induced dermatitis, with extracellular vesicles, in particular exosomes obtained from human cardiospheres or cardiosphere-derived cells. The present invention also provides a formulation comprising extracellular vesicles, in particular exosomes obtained from human cardiospheres or cardiosphere-derived cells, for use in the treatment of dermatitis, in particular radiation-induced dermatitis.

Abstract: The present invention relates to the identification, isolation, expansion and characterization of a specific type of multipotent adult cardiac stem cell. These adult stem cells are characterized in that they naturally express a specific pattern of markers, which can be used to assist with their isolation and expansion. In particular, the cells express SOX17 and GATA4, but do not express Oct4, Nanog, c-kit and telomerase reverse transcriptase. These cells are able to differentiate into one or more of the following cell types: adipocytes, osteocytes, endothelial cells and/or smooth muscle cells. They also display an unprecedented capacity for immunoregulation as well as providing, activating and/or inducing repair of damaged cardiac tissue. These adult stem cells may be used as therapeutic agents including, without limitation, for the regeneration of tissue, particularly for regeneration of damaged cardiac tissue, such as myocardium.

Abstract: The present invention relates to the identification, isolation, expansion and characterization of a specific type of multipotent adult cardiac stem cell. These adult stem cells are characterised in that they naturally express a specific pattern of markers, which can be used to assist with their isolation and expansion. In particular, the cells express SOX17 and GATA4, but do not express Oct4, Nanog, c-kit and telomerase reverse transcriptase. These cells are able to differentiate into one or more of the following cell types: adipocytes, osteocytes, endothelial cells and/or smooth muscle cells. They also display an unprecedented capacity for immunoregulation as well as providing, activating and/or inducing repair of damaged cardiac tissue. These adult stem cells may be used as therapeutic agents including, without limitation, for the regeneration of tissue, particularly for regeneration of damaged cardiac tissue, such as myocardium.

Abstract: The present invention relates to the identification, isolation, expansion and characterization of a specific type of adult cardiac stem cell. These adult stem cells are characterised in that they naturally express a specific pattern of markers, which can be used to assist with their isolation and expansion. The cells of the invention display an unprecedented capacity for providing, activating and/or inducing repair of damaged cardiac tissue. These adult stem cells may be used as therapeutic agents including, without limitation, for the regeneration of tissue, particularly for regeneration of damaged cardiac tissue, such as myocardium.