10 results about "Antisense nucleic acid" patented technology

The present invention relates to agents, compositions, and methods for treating or ameliorating symptoms of cancer. Exemplary synergistic therapies include the use of antisense oligonucleotide agents to suppress expression of TGF-β2, alone and in combination with interferon-gamma. One or more biomarkers can be used to select subjects for treatment.

This application provides antisense nucleic acid compounds that reduce the levels of certain nutrients in neurons of the central nervous system. UBE3A This application also provides compositions comprising antisense nucleic acid compounds and their application in the prevention or treatment of overdose in subjects. UBE3A Use in methods related to mRNA and / or UBE3A peptide levels.

Disclosed herein are systems for modifying nucleic acid splicing of a target RNA, e.g. SCN1A mRNA. The systems can be used in methods, e.g., methods of treating a genetic disorder, e.g., epilepsy. The systems can include an expression system or a modified small nuclear RNA (snRNA) that includes an exon splicing silencer (ESS) sequence and an antisense nucleic acid sequence that targets a target RNA.

This invention belongs to the field of liver disease research and biomedicine, specifically relating to the application of reagents targeting and inhibiting OXCT1 in combination with BHB or pharmaceutically acceptable salts thereof in the preparation of anti-HCC drugs. The reagents inhibiting OXCT1 include: siRNA, shRNA, antisense nucleic acid, and gene editing reagents that reduce OXCT1 expression; and small molecule inhibitors, peptides, antibodies, or fusion proteins that inhibit OXCT1 enzyme activity. This invention proposes a novel therapeutic strategy of targeting and inhibiting OXCT1 in combination with BHB. Studies have shown that targeting and inhibiting OXCT1 can block the pro-cancer metabolic pathway of BHB, reducing the effective concentration of BHB for its anti-cancer effect, enabling it to achieve significant anti-cancer effects at clinically tolerable concentrations and exert a synergistic anti-tumor effect. This invention solves the problem of limited clinical application of high-concentration BHB and provides a new combination therapy regimen with clinical translational potential for hepatocellular carcinoma.

The present invention provides an antisense oligomer having the base sequence depicted in SEQ ID NO: 26, an antisense oligomer having a base sequence resulting from substitution, deletion, insertion, or addition of 1 to 6 bases in the base sequence depicted in SEQ ID NO: 26, a pharmaceutically acceptable salt thereof, or a pharmaceutically acceptable hydrate thereof, an oligonucleotide conjugate in which the antisense oligomer is bound with a molecule capable of binding to an asialoglycoprotein receptor, and a pharmaceutical composition containing the same.

Modified antisense mucleic acid molecules ending with or without chain terminating bases targeting the HIV-1 viral genomic RNA 3′ LTR region, and uses thereof for inhibiting HIV-1 replication and infection, are disclosed. The antisense mucleic acid molecules more specifically target a sequence corresponding to about nucleotide 9628 to about nucleotide 9642 of HIV-1 clone pNL4-3.

The present disclosure relates to compositions and methods for treating liver cancers, especially hepatocellular carcinoma, using antisense (AS) nucleic acids directed against Insulin- like Growth Factor 1 Receptor (1GF-1R). The AS may be administered to the patients systemically, or may be used to produce an autologous cancer cell vaccine. In embodiments, the AS are provided in an implantable irradiated biodiffusion chamber comprising tumor cells and an effective amount of the AS. The chambers are irradiated and implanted in the abdomen of subjects and stimulate an immune response that attacks tumors distally. The compositions and methods disclosed herein may be used to treat many different kinds of liver cancer.