Abstract: The invention provides gene therapy vectors, such as adeno-associated virus (AAV) vectors, expressing a functional fragment of the miniaturized human micro-dystrophin gene and method of using these vectors to express the fragment of micro-dystrophin in skeletal muscles including diaphragm and cardiac muscle and to protect muscle fibers from injury, increase muscle strength and reduce and/or prevent fibrosis in subjects suffering from muscular dystrophy.

Abstract: Surface supported quantum wells with a confined surface state capture and stably confine neutral atoms and molecules in a nanometer precise environment. Depending on the physico-chemical conditions in the capturing process, the degree of occupancy, the temperature of the solid substrate, and/or the history of external stimuli like electromagnetic field pulses, these atoms, molecules or clusters assume unique configurations. The atoms or molecules are able to remain coupled to the quantum-well specific electronic state in the confinement and as such exhibit local and delocalized quantum entanglement. The capturing potential arises from the superposition of Pauli repulsion between the captured object and the quantum well-specific confined electronic state. This occurs within on-surface atomic or supramolecular assemblies or surface supported coordination or covalent networks.

Abstract: The invention provides gene therapy vectors, such as adeno-associated vims (AAV) vectors, expressing a functional fragment of the miniaturized human micro-dystrophin gene and method of using these vectors to express the fragment of micro-dystrophin in skeletal muscles including diaphragm and cardiac muscle and to protect muscle fibers from injury, increase muscle strength and reduce and/or prevent fibrosis in subjects suffering from muscular dystrophy.

Abstract: A method of producing a pluripotent stem cell is provided. The method is comprising contacting a non-pluripotent donor cell obtained from a mammalian donor with a compound characterized by general formulas (1) and (3). Furthermore, methods for inducing OCT4 and NANOG, increasing histone 3 lysine methylation and the maintenance of pluripotency are provided.

Abstract: The present invention provides novel HIV protease inhibitors containing substituted borane, carborane or metallacarborane clusters with 6 to 12 boron atoms in each cluster. The charge of each borane, carborane or metallacarborane cluster is 0, ?1 or ?2. The number of borane, carborane or metallacarborane clusters in the inhibitor molecule is 1 to 9, and the carborane clusters in metallacarborane inhibitors are coordinated to transition metal atom, selected from the group containing cobalt, iron, nickel and ruthenium. In the cluster, heteroatoms can be present, such as nitrogen, phosphorus, silicon, germanium, tin and sulphur. The present invention covers pharmaceutical compositions containing these inhibitors and their use, both in vitro and in vivo. They are characterized by high effectiveness and stability.

Abstract: Compositions and methods for suppressing the immune system of a mammal using ubiquitin and derivatives and analogs thereof.

Abstract: The present invention relates to methods for treating or preventing podocyte-related diseases and disorders using compounds that modulate the calcium sensing receptor or pharmaceutical compositions comprising thereof.

Abstract: Compositions and methods for suppressing the immune system of a mammal using ubiquitin and derivatives and analogs thereof.

Abstract: This invention relates to a nucleic acid that contains at least one nucleic acid sequence coding for a polypeptide, polypeptide being capable of reducing the enzymatic activity of an invertase; the polypeptide itself; and transgenic plants that contain this nucleic acid sequence. The invention further relates to methods of preparing such transgenic plants having reduced storage sucrose loss.

Abstract: Transgenic mice carrying a transgene comprising a nucleic acid molecule encoding protein useful for regulating the expression of genes in eukaryotic cells in a highly controlled manner are disclosed. In the regulatory system of the invention, transcription of a tet operator-linked nucleotide sequence is stimulated by a transcriptional activator fusion protein composed of two polypeptides, a first polypeptide which binds to tet operator sequences in the presence of tetracycline operatively linked to a second polypeptide activates transcription in eukaryotic cells. In a preferred embodiment, the transgene encoding the transcriptional activator fusion protein is integrated at a predetermined location within the chromosome of the transgenic mouse.