Abstract: The present invention is directed to an expression vector and a composition comprising same, wherein the expression includes a precursor of miR-193 a. Further provided are methods of using the expression vector of the invention or the composition comprising same, such as in diagnosing and/or treating head and neck squamous cell carcinoma, in a subject in need thereof.

Abstract: Micro-organ compositions and methods of implanting into the central nervous system (CNS) for the treatment of CNS-related diseases are encompassed. Specifically, the disclosure provides methods for treating disorders including cancer and lysosomal storage diseases, the methods comprising implanting a micro-organ into the CNS, wherein the micro-organ secretes a recombinant protein, and wherein the micro-organ is maintained in the CNS, and secretes protein, for at least seven days.

Abstract: Provided herein is a genetically-modified micro-organ that provides a sustained delivery of a therapeutic peptide. The genetically-modified micro-organ may comprise a viral vector or expression cassette comprising at least two nucleic acid sequences encoding the therapeutic peptide separated by a cleavable linker. Further provided herein is a method of treating or preventing a disease or disorder in a human subject that can be treated or prevented by administration of a therapeutic peptide over a sustained time period using the genetically-modified micro-organ described herein.

Abstract: The present invention is directed to long-lasting therapeutic formulations and their methods of use wherein the formulation comprises a genetically modified micro-organ that comprises a nucleic acid sequence operably linked to one or more regulatory sequences. The present invention is further directed to methods providing sustained expression of therapeutic polypeptides and prolonged therapeutic effects, such as erythropoietin and interferon.

Abstract: An isolated polypeptide comprising, contiguously, a first amino acid sequence being at least 90% homologous to amino acid coordinates 1-398 of SEQ ID NO: 2, a tryptophan residue and a second amino acid sequence being at least 90% homologous to amino acid coordinates 400-523 of SEQ ID NO:2. Also provided are polynucleotide sequences encoding this polypeptide and uses of same in the treatment of medical conditions associated with ion channel insufficiency.

Abstract: An isolated polypeptide comprising, contiguously, a first amino acid sequence being at least 90% homologous to amino acid coordinates 1-398 of SEQ ID NO: 2, a tryptophan residue and a second amino acid sequence being at least 90% homologous to amino acid coordinates 400-523 of SEQ ID NO: 2. Also provided are polynucleotide sequences encoding this polypeptide and uses of same in the treatment of medical conditions associated with ion channel insufficiency.