42 results about "Gene Therapy Agent" patented technology

A system and method for optimizing the systemic delivery of growth-arresting lipid-derived bioactive drugs or gene therapy agents to an animal or human in need of such agents utilizing nanoscale assembly systems, such as liposomes, resorbable and non-aggregating nanoparticle dispersions, metal or semiconductor nanoparticles, or polymeric materials such as dendrimers or hydrogels, each of which exhibit improved lipid solubility, cell permeability, an increased circulation half life and pharmacokinetic profile with improved tumor or vascular targeting.

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.

The present invention provides for improved vectors for use in gene therapy. Utilizing the cancer specific DF3 / MUC1 promoter to drive a replication essential gene, vectors are made conditionally replication-competent, permitting wider infection and expression of tumor cells. In addition, therapeutic genes and adjunct therapies further increase anti-tumor efficacy.

The present invention provides methods for enhanced delivery of various therapeutic agents, such as gene therapy agents, to the vasculature of a target organ in a mammalian subject. The methods for targeted gene therapy in the mammalian liver as a whole, or in a single hepatic lobe, are disclosed. The disclosed methods rely on minimally invasive catheter-based procedures wherein a target organ is isolated and treated locally with a gene therapy agent. The methods offer more efficient and localized transfection of tissue and are well-suited for gene therapy in human subjects.

The present invention relates to novel vectors, to DNA vaccines and gene therapeutics containing said vectors, to methods for the preparation of the vectors and DNA vaccines and gene therapeutics, and to therapeutic uses of said vectors. More specifically, the present invention relates to novel vectors comprising an expression cassette of a gene of a nuclear-anchoring protein, which contains a DNA binding domain capable of binding to a specific DNA sequence and a functional domain capable of binding to a nuclear component and a multimerized DNA sequence forming a binding site for the nuclear-anchoring protein, and optionally an expression cassette of a gene, genes or a DNA sequence or DNA sequences of interest. The present invention further relates to DNA vaccines and gene therapeutics containing the novel vectors, to methods for the preparation of the novel vectors and the DNA vaccines and gene therapeutics containing the novel vectors, and to the use the vectors in therapy.

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.

The present invention relates to novel vectors, to DNA vaccines and gene therapeutics containing said vectors, to methods for the preparation of the vectors and DNA vaccines and gene therapeutics containing the vectors, and to therapeutic uses of said vectors. More specifically, the present invention relates to novel vectors comprising (a) an expression cassette of a gene of a nuclear-anchoring protein, which contains (i) a DNA binding domain capable of binding to a specific DNA sequence and (ii) a functional domain capable of binding to a nuclear component and (b) a multimerized DNA sequence forming a binding site for the anchoring protein, and optionally (c) one or more expression cassettes of a DNA sequence of interest. In particular the invention relates to vectors that lack a papilloma virus origin of replication. The nuclear-anchoring protein might be the E2 protein of Bovine Papilloma Virus type 1 or Epstein-Barr Virus Nuclear Antigen 1. The invention also relates to vectors that lack an origin of replication functional in a mammalian cell. The invention further relates to methods for expressing a DNA sequence of interest in a subject.

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.

The present invention relates to cardiac performance, in particular to regulating cardiac performance via recombinant troponin I (TnI) protein and nucleic acids encoding recombinant TnI. The present invention provides nucleic acids encoding gain of function TnI proteins (e.g., cTnlA164H), vectors containing such nucleic acids, host cells containing such vectors, transgenic animals carrying a gain of function TnI protein (e.g., a cTnlA164H transgene), and therapeutic agents (e.g., comprising recombinant TnI, TnI analogues, synthetic TnI, or the like) or agents for gene therapy of heart failure or disease for research and therapeutic uses.

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 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 ars 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.

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.

A system and method for optimizing the systemic delivery of growth-arresting lipid-derived bioactive drugs or gene therapy agents to an animal or human in need of such agents utilizing nanoscale assembly systems, such as liposomes, resorbable and non-aggregating nanoparticle dispersions, metal or semiconductor nanoparticles, or polymeric materials such as dendrimers or hydrogels, each of which exhibit improved lipid solubility, cell permeability, an increased circulation half life and pharmacokinetic profile with improved tumor or vascular targeting.

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.

The present invention provides methods for enhanced delivery of various therapeutic agents, such as gene therapy agents, to the vasculature of a target organ in a mammalian subject. The methods for targeted gene therapy in the mammalian liver as a whole, or in a single hepatic lobe, are disclosed. The disclosed methods rely on minimally invasive catheter-based procedures wherein a target organ is isolated and treated locally with a gene therapy agent. The methods offer more efficient and localized transfection of tissue and are wellsuited for gene therapy in human subjects.

The invention relates to site-specific integrating expression vectors for therapy Hemophilia B and to methods of preparing them. A vector of the invention contains a human Factor IX gene in a vector constructed using as a chromosome targeting sequence a polynucleotide without any important physiological function-related gene homologous to DNA on the short arms of human group D and human group G chromosomes. The vector of the invention provides high stability of Factor IX expression, high expression efficiency, no immunogenicity and safety in use.

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 present invention relates to the compositions for a gene therapy of rheumatoid arthritis including a gene encoding an anti-angiogenic protein or parts thereof. More specifically, the present invention provides a gene therapy of rheumatoid arthritis by preparing a recombinant vector that expresses a gene encoding an anti-angiogenic protein such as angiostatin or parts thereof, and transplanting the recombinant vector or a cell that is transfected or transduced with the recombinant vector into the affected area of a patient, and also provides the compositions for the gene therapy. The compositions for the gene therapy according to the present invention can be used effectively for the treatment of rheumatoid arthritis, for which effective treating methods have not been developed until now, by providing the anti-angiogenic proteins into the knee of a patient continuously to prevent the synovial tissue hyperplasia and the resulting inflammation.

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 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.

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.

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.