Improved compositions and methods for treating muscular dystrophy by administering antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon skipping are described.

The present invention relates to peptides, proteins, nucleic acids and cells for use in immunotherapeutic methods. In particular, the present invention relates to the immunotherapy of cancer. The present invention furthermore relates to tumor-associated T-cell peptide epitopes, alone or in combination with other tumor-associated peptides that can for example serve as active pharmaceutical ingredients of vaccine compositions that stimulate anti-tumor immune responses, or to stimulate T cells ex vivo and transfer into patients. Peptides bound to molecules of the major histocompatibility complex (MHC), or peptides as such, can also be targets of antibodies, soluble T-cell receptors, and other binding molecules.

The oligonucleotide compositions of the present invention make use of combinations of oligonucleotides. In one aspect, the invention features an oligonucleotide composition including at least 2 different oligonucleotides targeted to a target gene. This invention also provides methods of inhibiting protein synthesis in a cell and methods of identifying oligonucleotide compositions that inhibit synthesis of a protein in a cell.

The present invention relates to the field of fibrosis and inflammation and more particularly to the use of ADAM12 (A Disintegrin and Metalloproteinase 12) inhibitors to prevent or treat inflammation-induced fibrosis. The present invention also relates to the use of ADAM12 as a marker for inflammation-induced fibrosis and to the ablation of ADAM12 expressing cells as therapeutic approach to interfere with the development of pro-fibrotic cells.

Antisense polynucleotides and their use in pharmaceutical compositions to induce exon skipping in targeted exons of the gamma sarcoglycan gene are provided, along with methods of preventing or treating dystrophic diseases such as Limb-Girdle Muscular Dystrophy.

Disclosed are improved shRNA molecules, termed “organic shRNA” (OshRNA), that incorporate certain structural features that increase the likelihood that the desired guide strand is produced while reducing accumulation of passenger strands that might contribute to off-target effects. Also provided herein are nucleic acids encoding OshRNAs, kits, cells, and transgenic animals comprising such nucleic acids, as well as methods of making and using OshRNAs and/or nucleic acids encoding OshRNAs.

The present invention relates to RNAi constructs for reducing expression of the MARC1 gene. Methods of using such RNAi constructs to treat or prevent liver fibrosis and fatty liver diseases, such as nonalcoholic fatty liver disease and nonalcoholic steatohepatitis, are also described.

Described herein are methods and compositions for the treatment of colon cancers.

The present invention relates to methods and compositions comprising an inhibitor of KLF11 signaling for treatment of gastrointestinal motility disorders, obesity and diabetes.

The present invention provides a nucleic acid having activity to suppress expression of β2GPI, a pharmaceutical composition comprising the nucleic acid, and a prophylactic or therapeutic drug containing the nucleic acid for autoimmune diseases such as APS, SLE and the like and thrombosis in hemodialysis.