Provided are methods related generally to the fields of molecular biology and virology, in particular the use of immunosuppressive agents and methods for treating using gene therapy, notably Duchenne muscular dystrophy.

In certain embodiments, hybridization results in modulation of the amount activity or expression of the target nucleic acid in a cell. In certain embodiments, the target nucleic acid is a nucleic acid that encodes cartilage oligomeric matrix protein. In certain embodiments, the target nucleic acid is a nucleic acid expressed in the growth plate, tendon, or cartilage.

Methods and composition for modulating a target genome and stable integration of a transgene of interest into the genome of a cell are disclosed.

The described invention provides a method of treating a patient with an epithelial cancer comprising administering a composition comprising a therapeutic amount of an inhibitor of a BTK protein and one or more chemotherapeutic agent(s) selected from the group consisting of an antimetabolite, a platinum coordination compound, an alkylating agent and a combination thereof, wherein the composition is effective to reduce one or more of tumor cell growth, tumor cell clonogenicity, tumor cell proliferation, tumor cell viability and tumor volume and the therapeutic amount of the inhibitor of a BTK protein and the one or more chemotherapeutic agent(s) exerts a synergistic effect. The described invention also provides methods of treating a chemotherapy drug-resistant cancer and sensitizing a cancer patient to chemotherapy.

The present disclosure provides for methods and compositions for treating cancer. A subject having lymphoma is administered an EZH2 inhibitor and an HDAC inhibitor. The combination of the EZH2 inhibitor and the HDAC inhibitor produces a synergistic effect on the cancer compared to the effect of the EZH2 inhibitor or the HDAC inhibitor alone.

Provided herein are RNA aptamers targeting CD5L. Further provided herein are methods of use thereof for the treatment of a disease or disorder, such as cancer.

Cystic fibrosis (CF) is a genetic disease caused by mutations in the gene encoding the 5 CFTR Cl— channel and affects several organs, but the most severe consequences are observed in the lung. The inventors have now instigated the combination of Orkambi® and ANO1 TSB (SEQ ID NO:1) and show that said combination increases mucociliary clearance and chloride channel activity. Thus the combination represents an alternative treatment for CF subjects.

Described herein are antisense inhibitor adjuvant compositions comprising modified nucleotide sequences complementary to a Homo sapiens interleukin 10 (IL-10) or interleukin 10 receptor alpha (IL-10RA) genes and methods for use thereof.

The present invention relates to methods for treating and preventing ophthalmological disease and disorders, comprising administering Antagonist A or another pharmaceutically acceptable salt thereof, optionally in combination with another treatment, to a subject in need thereof. The present invention also relates to methods for treating and preventing ophthalmological disease and disorders, comprising administering an anti-C5 agent (e.g., ARC1905), optionally in combination with another treatment, to a subject in need thereof.

Embodiments of the disclosure include methods and compositions for in situ cardiac cell regeneration, including transdifferentiation of cardiac cells to cardiomyocytes. In particular embodiments, in situ cardiac cell regeneration encompasses delivery of p63 shRNA and one or both of Hand2 and myocardin, and in specific embodiments further includes one or more of Gata4, Mef2c, and Tbx5. In specific aspects of the disclosure, adult cardiac fibroblasts are reprogrammed into cardiomyocytes using viral vectors that harbor p63 shRNA and one or both of the transcription factors Hand2 and myocardin.