13 results about "Gene Therapy Agent" patented technology

The present invention relates to gene therapy agents for the treatment of pulmonary alveolar proteinosis (PAP), particularly autoimmune PAP (aPAP). In particular, the present invention relates to gene therapy vectors which drive transient and / or low-level expression of granulocyte-macrophage colony-stimulating factor (GM-CSF), which provide a therapeutic effect without therapy-associated toxicity. The invention further relates to related products and an animal model of aPAP.

The present invention relates to a base correction system that corrects mitochondrial DNA mutations G3460A, G11778A, or T14484C, which are present in patients with Leber's hereditary optic neuropathy (LHON), to a normal genotype. Specifically, the present invention provides a base editor capable of correcting a mutation site in a mitochondrial gene of an LHON patient to a normal genotype. The present invention also provides a method for correcting a mitochondrial gene mutation using a fusion protein or a polynucleotide encoding such a fusion protein that recognizes a specific site in the mitochondrial gene of an LHON patient and specifically corrects the adenine base at position 3460, the adenine base at position 11778, or the cytosine base at position 14484. The base editor or polynucleotide according to the present invention can be used in cells or in an extracellular test tube environment to correct DNA mutations specifically expressed in LHON, and more preferably, can be used as a gene therapy agent to prevent or treat the disease. Thus, the present invention also provides a use of the substance for preventing or treating Leber's hereditary optic neuropathy.

The invention discloses a pH and enzyme dual-response type targeted DNA nano-carrier for relieving tumor cell hypoxia as well as a preparation method and application of the pH and enzyme dual-response type targeted DNA nano-carrier. The DNA nano-carrier comprises m tandem repeat units, each tandem repeat unit comprises a long single-stranded DNA repeat unit, three complementary short single-stranded DNAs and siRNA, each of the three complementary short single-stranded DNA sequences comprises a functional region and a base complementary region, and the functional region is a pH-responsive nucleic acid complementary sequence or a nucleic acid aptamer sequence for specifically targeting cancer cells; siRNA is a gene therapeutic agent for relieving tumor hypoxia, an extended positive-sense strand of siRNA comprises a 5 '-terminal extended pH response sequence and a positive-sense strand, and the 5'-terminal extended pH response sequence is complementary with a pH-responsive nucleic acid complementary sequence of the complementary short single-stranded DNA. The nano-drug carrier has excellent drug loading capacity and targeting property, can deliver anti-cancer drugs such as siRNA in a targeted manner, responds to a slightly acidic / enzyme environment of tumor cells and is quickly released, and the anti-tumor treatment effect is improved.

The present invention relates to: an antisense oligonucleotide (ASO) targeting an FKS1 gene, an FKS2 gene, a CHS3 gene and / or a GWT1 gene of a Candida sp. fungus; an antibacterial composition comprising same; and a pharmaceutical composition for preventing or treating diseases caused by pathogenic fungi. The ASO according to the present invention can be used as a gene therapeutic agent having a fungus-specific antibacterial effect by specifically inhibiting FKS1, FKS2, CHS3 and / or GWT1 genes, and can be a useful gene therapeutic agent that can be used in combination with or replace conventional antifungal agents for antifungal agent-resistant fungi or multidrug-resistant fungi.

The present invention relates to polymer-based nucleic acid molecule (e.g., mRNA) transporters having ionizable moieties. More specifically, the present invention provides novel polymers that can transport any negatively charged dielectric substance to a desired site, depending on the length of the monomer and polymer. The polymers of the present invention can be effectively used in gene therapy agents or vaccines containing genetic material, such as mRNA, for therapeutic or prophylactic purposes.

The present invention provides gene or RNA therapeutic vectors for use in the treatment of non-malignant CNS disorders, which are to be injected into the pons by convection-enhanced delivery (CED). The invention also provides a method for treating non-malignant CNS disorders, comprising injecting a gene or RNA therapeutic vector into the pons of a subject in need (CED), and a method for distributing a gene therapy agent throughout the nervous system, wherein a viral vector transported anterograde and retrogradely is injected into the pons and middle cerebellar peduncle of a subject from a cannula inserted from the cerebellum and middle cerebellar peduncle into the pons.

The invention provides an oligonucleotide hydrogel micro-nano gene compound as well as a preparation method and application thereof, and belongs to the technical field of biological medicines. The preparation method comprises the following steps: firstly, designing C5ASO, and loading the C5ASO on a nano-carrier modified with HAP-1 peptide (targeting fibroblast-like synovial cells) and active ester; meanwhile, the porous HAMA hydrogel microspheres are prepared by utilizing a photo-crosslinking micro-fluidic technology; then, DBCO-TIMP and an azide group are respectively introduced into the liposome and the hydrogel microspheres through Michael addition and an amide coupling reaction, so that a micro-nano gene compound (C5ASO (at) HAP-CL-TIMP (at) HMs) is formed; in addition, the liposome and the hydrogel microspheres are efficiently compounded through click chemistry. The hydrogel micro-nano gene compound not only can efficiently load ASO lipidosome, but also can enhance the stability of a gene therapeutic agent in an in-vivo delivery process. Meanwhile, the MMP-9 response peptide structure in the RA articular cavity inflammation microenvironment can be accurately activated, so that continuous complement intervention is realized.

The present invention relates to a gene therapy method capable of continuously and efficiently correcting genes, and a gene therapy product using same. It is expected that the gene therapy product of the present invention can present clues for gene therapy for muscular diseases including Duchenne muscular dystrophy.

Compositions and methods of use characterized by nucleic acids and vectors. In particular, the present disclosure features nucleic acid compositions and methods relating to temporally modulating expression of gene therapeutics comprising a therapeutic transgene. The disclosed compositions, methods, and uses relate to gene and cell therapy.

Featured are compositions and methods of use in conjunction with gene therapy agents. In particular, the disclosure features compositions and methods designed to permanently and / or site- specifically alter expression of a gene therapy agent comprising a therapeutic transgene.

The present invention relates to a gene therapy method capable of continuously and efficiently correcting genes, and a gene therapeutic agent using same. The gene therapeutic agent of the present invention is expected to be able to treat genetic diseases including Duchenne muscular dystrophy and cystic fibrosis, and furthermore, to provide therapeutic cues for a wide range of diseases caused by genetic defects.

The present disclosure relates to a dual helper plasmid for producing a recombinant adeno-associated virus. A double transfection method using the dual helper plasmid of the present disclosure is advantageous over the triple transfection method typically used for production of adeno-associated virus in terms of 1) increased chance of co-transfection, 2) increased productivity of recombinant adeno-associated virus, 3) reduction in cost and time of plasmid production and purification, etc., and thus can be usefully utilized for effective production of a gene therapy agent.

The present invention relates to multi-layer nanoparticles for nucleic acid delivery, a method for preparing same, a nucleic acid delivery composition comprising same, an antibacterial composition, and a pharmaceutical composition for preventing or treating infectious diseases. The multi-layer nanoparticles for nucleic acid delivery comprise: gold nanoparticles; a polyethyleneimine modified on the surface of the gold nanoparticles; and chitosan applied on the gold nanoparticles modified with the polyethyleneimine. According to the present invention, the introduction of nucleic acids (e.g., ASOs) into target bacterial cells is maximized, and highly efficiently loaded nucleic acids can be effectively delivered into bacteria to maximize the effect of inhibiting target nucleic acids, and thus the present invention can be useful as an antibacterial agent and a gene therapeutic agent.