41 results about "Gene Therapy Agent" patented technology

The present invention is directed to novel nucleic acid molecules and methods for their use. More specifically, the novel nucleic acid molecules of the present invention are capable of tightly and specifically interacting with a target molecule of interest not only through standard Watson-Crick base pairing, but also through additional features which allow the antisense molecules to become topologically “locked” onto the target nucleic acid, thereby imparting improved transcription and translation inhibitory properties.

A unique transcription product, CRCA-1, and alternative translation products generated therefrom, are disclosed. The transcript and its translation products are markers for colorectal cells. Screening and diagnostic reagents, kits and methods for metastasized colorectal cancer are disclosed as are reagents, kits and methods for identifying adenocarcinomas as colorectal in origin. Compounds, compositions and methods of treating patients with metastasized colorectal cancer and for imaging metastasized colorectal tumors in vivo are disclosed. Compositions and methods for delivering active compounds such as gene therapeutics and antisense compounds to colorectal cells are disclosed. Vaccines compositions and methods of for treating and preventing metastasized colorectal cancer are disclosed.

Disclosed is a siRNA-encapsulating polymeric micelle complex (a polyion complex) having high monodispersibility and structural stability and excellent in the ability of transporting siRNA into a cell. Further disclosed are a nucleic acid delivery device, a nucleic acid delivery kit, a pharmaceutical composition, and a gene therapy agent, each of which uses the complex. The polyion complex is characterized by comprising: a block copolymer composed of a polyethylene glycol moiety and a polycation moiety having a thiol group as a side chain at the terminus; and siRNA.

The present invention relates to methods and devices for administration of vaccines and gene therapeutic agents into the intradermal layer of skin.

Gene therapeutics to be used in treating diseases showing sensitivity to gene therapy, characterized by containing as the active ingredient an efficacious amount of a functional substance which has a function of having an affinity for a virus containing a gene usable in the gene therapy and another function of having an affinity specific for a target cell with a need for the gene transfer, or an efficacious amount of a functional substance which has an affinity for the above virus and an efficacious amount of another functional substance which has an affinity specific for the above cell.

The present invention relates to an anticancer or an anti-metastatic agent for gene therapy, more precisely, an anticancer or an anti-metastatic agent for gene therapy containing a gene carrier or cells harboring human apolipoprotein (a) kringle KIV9-KIV10-KV (LK68) or KV (LK8) gene as an effective ingredient, and a treatment method for cancer using the same. The agent for gene therapy of the present invention has an inhibiting effect on the growth and the metastasis of a tumor, so it can be effectively used for the prevention and the treatment of various solid tumors as a metastasis inhibitor or a therapeutic agent for primary tumors.

A super-antigen immune gene treater for liver cancer is characterized by that the liver cancer cell specific regulation sequence mediated super-antigen moleculae and co-stimulation moleculae are target expressed in liver cancer cells to specifically enhance the immunogenicity of cancer cell and the recognition action to immunoeffect cells. after the treater is injected to cancer region, it can effectively kill cancer cells.

A nanohybrid of the potent gene therapeutic agent siRNA (small interfering RNA) and a target-specific layered inorganic hydroxide, a preparation method thereof, and a pharmaceutical composition for tumor treatment containing the target-specific, siRNA/layered inorganic hydroxide nanohybrid. The nanohybrid increases the in vivo stability of the siRNA, and a target-specific multifunctional ligand, which is bonded to the layered inorganic hydroxide and can bind specifically to a tumor, increases the efficiency of tumor-specific transfer of the siRNA such that the siRNA shows tumor therapeutic activity even at a relatively low dose. Thus, the nanohybrid will be widely useful for target-specific antitumor therapies.

A method and gene therapy agent for treating a vulnerable plaque associated with a blood vessel of a patient is disclosed. The method includes providing at least one gene therapy agent encoding at least one protein. The gene therapy agent is administered to a target cell population. The protein is expressed within the patient from a portion of the target cell population. The vulnerable plaque is modified as a result of the protein expression. The gene therapy agent includes at least one polynucleic acid encoding at least one protein. Administration of the gene therapy agent to a target cell population results in expression of the protein capable of modifying the vulnerable plaque.

The present invention relates to new NOS variants or mutants which contain structural alterations in the site of Akt dependent phosphorylation. The altered NOS proteins or peptides, especially the human eNOS proteins or peptides, Akt proteins or polypeptides and their encoding nucleic acid molecules are useful as gene therapy agents for the treatment of diseases including post angioplasty restenosis, hypertension, atherosclerosis, heart failure, diabetes and diseases with defective angiogenesis. NOS proteins and peptides are also useful in methods of screening for agents which modulate NOS activity.

In some aspects, the disclosure relates to compositions and methods for modulating ( e.g., increasing and/or decreasing) bone mass in a subject. In some aspects, the disclosure provides isolated nucleic acids, and vectors such as rAAV vectors, configured to express transgenes that promote (e.g., increase) or inhibit (e.g., decrease) activity, differentiation, or function of certain types of bone cells, for example osteoblasts, osteoclasts, osteocytes, etc. In some embodiments, the isolated nucleic acids and vectors described by the disclosure are useful for treating disorders and conditions associated with increased bone mass (e.g., osteopetrosis) or decreased bone mass (e.g., osteoporosis).

The invention deals with the gene drugs for therapy Hemophilia B and its method of preparation. It contains human source gene vector-FIX recombinant constructed using DNA sequence as leading sequence of therapy gene without important physiological function-related gene on the short arms of human group D,G chromosomes or to which DNA sequence is homologous. This invention also provides the method of preparing gene drug. The therapy gene of gene drug for hemophilia B has a high stability of expression, safety, high expression efficiency and no immunogenecity.

The present invention relates to a gene therapeutic agent using truncated soluble cDNA of VEGF receptor protein (VEGFR) and adeno-associated virus(AVV) system, and more particularly, relates to a gene therapeutic agent specific to large intestine cancer, bladder cancer and/or lung cancer, comprising a rAAV vector containing truncated soluble cDNA of VEGFR, and a rAAV vector containing the said vector and VEGF-A anti-sense cDNA. The gene therapeutic agent according to the present invention reduces the growth of tumors by inhibiting the expression and function of VEGF involved in angiogenesis necessary for the proliferation and metastasis of tumors. Thus, the inventive gene therapeutic agent can be effectively used to treat cancer at a gene level.

Disclosed herein is a gene therapeutic agent and pharmaceutical composition for the prevention and treatment of lung cancer. For aerosol delivery, chemically synthesized polyester amine is used as a carrier in the gene therapeutic agent. The polyester amine/Akt1 siRNA complex is found to be effectively delivered to the lungs of K-ras null mice through a nose-only inhalation system and to significantly suppress lung cancer progression as denoted by gene delivery efficiency and inhibition of Akt-related signals and cell cycle. Thus, the aerosol delivery of polyester amine-mediated Akt1 siRNA is provided as an effective model for noninvasive gene therapy.

The invention discloses application of a sodium ion channel blocker in the preparation of drugs to treat melanoma. The sodium ion channel blocker is made with any one or two of tetrodotoxin and dehydrated tetrodotoxin. The invention also provides a drug to treat melanoma; the drug comprises the sodium ion channel blocker, a second therapeutic agent and a pharmaceutically acceptable carrier; the second therapeutic agent includes one or more of a chemotherapeutic agent, an immunotherapeutic agent, a gene therapeutic agent and a radiotherapeutic agent. The sodium ion channel blocker can inhibit the proliferation and tumorigenesis of melanoma cells, and can cooperate with the second therapeutic agent, especially mitogen-activated extracellular signal regulation kinase inhibitor, to arrive at better treatment of melanoma.

The present invention relates to a complex of an antitumor adenovirus coated with ErbB-PEG-PAMAM and the use thereof. When using the complex of the present invention, a tumor cell-specific gene transfer effect by an excellent tumor cell infiltration ability is excellent, hepatotoxicity is low, a blood flow retention time is prolonged. Thus, the complex of the present invention can be used as a pharmaceutical composition for the treatment of a tumor and a gene therapy agent which can be administered systemically.