Abstract: The present invention relates to methods for restoring the function of a mutated dysferlin comprising the step of preventing splicing of one or more exons which encode amino acid sequences that cause said dysferlin dysfunction. Particularly, the splicing of exon 32 is prevented. The present invention also relates to a method for treating a dysferlinopathy in a patient in need thereof, comprising the step of administering to said patient antisense oligonucleotides complementary to nucleic acid sequences that are necessary for correct splicing of one or more exons which encode amino acid sequences that cause said dysfunction. Particularly, the splicing of exon 32 is prevented.

Abstract: A pharmaceutical and a method for preventing and treating VHSV infection in fish with a VHSV-binding aptamer is provided. Also provided are a method for removing VHSV from an area of water and a method and kit for detecting VHSV with high sensitivity using a VHSV-binding aptamer.

Abstract: Long non-coding RNAs (lncRNAs) and methods of using them diagnostically and therapeutically for treatment of cancer, stem cell therapy, or regenerative medicine are disclosed. In particular, the invention relates to lncRNAs that play roles in regulation of genes involved in cell proliferation, differentiation, and apoptosis. Such lncRNAs can be used as biomarkers to monitor cell proliferation and differentiation during cancer progression or tissue regeneration. One of the identified lncRNAs, referred to as PANDA (a P21-Associated NcRNA, DNA damage Activated), inhibits the expression of apoptotic genes normally activated by the transcription factor NF-YA. Inhibitors of PANDA sensitize cancerous cells to chemotherapy and can be used in combination with chemotherapeutic agents for treatment of cancer.

Abstract: The disclosure is directed to methods of treating hematologic malignancies. More particularly, the disclosure is directed to methods of treating hematologic malignancies using Rho kinase (ROCK) inhibitors and myosin light chain-specific inhibitory RNA molecules. The disclosure is further directed to methods of identifying drug candidates for inhibiting ROCK in hematologic malignancies.

Abstract: TLR3 agonist compounds, compositions and methods are provided for stimulating the activity of TLR3. The compositions comprise oligonucleotide-based compounds that bind to and activate TLR3. The compositions may also comprise oligonucleotide-based compounds that bind to and activate TLR3 in combination with other therapeutic and/or prophylactic compounds and/or compositions. Methods of using these compounds and compositions for stimulation of TLR3 activity and for prevention or treatment of diseases wherein modulation of TLR3 activity would be beneficial are provided.

Abstract: The preset invention relates to a biomarker for identifying the subgroup of early-stage lung adenocarcinoma patients in early-stage non-small cell lung cancer (NSCLC), which is T-lymphokine-activated killer cell-originated protein kinase (TOPK), and a therapeutic target for lung cancer.