159results about "Apolipeptides" patented technology

To provide a gene therapy vector comprising an expression cassette coding for a mammalian apolipoprotein E (APOE); a pharmaceutical composition; and a method for preventing, inhibiting or treating Alzheimer's disease in mammals.SOLUTION: The invention provides a gene therapy vector comprising an expression cassette coding for a mammalian apolipoprotein E that has a residue other than arginine at at least one of positions 112, 136 or 158, but is not a mammalian apolipoprotein E that has R112, R136 and R158 or a mammalian apolipoprotein E that has C112, R136 and C158, or coding for an antibody that binds to APOE4 or disrupts the binding of APOE to heparan sulfate proteoglycans.SELECTED DRAWING: None

Described herein are compositions and methods for treatment of Age-related Macular Degeneration (AMD). In particular, described herein are polypeptides comprising helical structures having ATP binding cassette transporter membrane stabilization and agonist activity, transporter protein binding activity, and / or that may result in a cholesterol efflux, as well as methods of using these peptides to treat AMD.

The present invention relates to high density lipoprotein (HDL) or HDL mimetics for use in the prevention and / or treatment of graft-versus-host disease (GvHD) or cytokine release syndrome in a subject.

Methods of treating age-related macular degeneration (AMD) may include the administration of amphipathic, ATP binding cassette transporter membrane agonists to reduce the amount of drusen in the subject in need thereof. These methods may be particularly useful for treatment of intermediate AMD.

The purpose of the present invention is to provide a therapeutic agent for dilated cardiomyopathy disease which produces excellent effects in the treatment of dilated cardiomyopathy (DCM) and provides a given benefit to a large number of patients. The present invention is a therapeutic agent for dilated cardiomyopathy comprising at least one selected from the group consisting of mesenchymal stem cell and microparticles derived from the mesenchymal stem cell. It is preferable that the mesenchymal stem cell is a cell having the ability to contain or secrete microparticles, that the microparticles have an average particle size of 1000 nm or less, and that they are exosomes.

The invention relates to the technical field of biology, in particular to a promoter with real skin specificity and application thereof. The promoter provided by the invention is selected from a promoter of any one or more of the following genes: GPN, FOS or APO. The promoter with true skin specificity provided by the invention can specifically express a target gene in dermis and remove off-target expression of liver and subcutaneous lipid membrane muscle at the same time, the promoter sequence is shorter than that of a broad-spectrum promoter, and the human hGPN promoter can be more safely applied clinically.

The invention relates to a bacterial strain containing an open reading frame which codes for a recombinant protein, thereby controlling a functional promoter. The invention is characterized in that the bacterial strain contains an open reading frame which codes for a muted peptidoglycan-associated lipoprotein (PAL protein), thereby controlling a functional promoter, wherein the PAL protein is muted such that the PAL protein does not contain a membrane anchor for the outer cell membrane of the bacterium. The invention additionally relates to a plasmid which codes for a recombinant protein and the muted PAL protein and to a method for the fermentative production of recombinant protein using the bacterial strain according to the invention. In this manner, product yields in the culture residue which are increased compared to the prior art can be achieved without leading to a substantial die-off of the bacterial cells.

To provide a new immunotherapeutic agent useful for treating obesity.SOLUTION: The pharmaceutical composition contains a peptide having a specific amino acid sequence containing a B cell epitope. Preferably the composition includes an aluminium salt (Alhydrogel (Al (OH) 3) and monophosphoryl lipid A as adjuvants. Intramuscular administration of the composition to a subject results in the production of antibodies capable of binding to ApoB-100 in vivo, and the antibodies exert a therapeutic effect on obesity.SELECTED DRAWING: Figure 14

A method of treating leukocytosis, endothelial dysfunction, and carditis comprising administering to a subject a lipid-binding protein-based conjugate.

The invention relates to modified apolipoprotein with a targeting body. The targeting body may for example be an antibody or antigen binding fragment that allows targeting of e.g. a specific cell, tissue or organ. The modified apolipoprotein can be used as a carrier for a payload as such or when incorporated in a lipid nanoparticle. The modified apolipoprotein finds use in the treatment or prevention of diseases, or targeting a pay load to a specific target site.

The invention relates to an APOE typing and quantitative analysis method, and belongs to the technical field of molecular diagnosis. The invention provides a method for detecting the subtype and expression quantity of apolipoprotein E (APOE) in a simply treated plasma or tissue sample by using mass spectrometry. According to the method, a plurality of APOE protein subtype common polypeptides are used as quality control anchor points, each subtype characteristic polypeptide is calibrated, the detection stability and reliability of the characteristic polypeptides can be effectively analyzed, and the APOE subtypes can be accurately qualitative and quantitative at the same time. The method has the advantages of high efficiency, accuracy, stability and the like, and has a wide clinical application prospect.

Provided herein are gene editing systems and compositions directed to effectuate in vivo edits in the LPA gene. Treatment or prevention of cardiovascular disease through disruption of the production of apo(a) through genetic editing and the reduction of the blood lipoprotein(a) [Lp(a)] concentration is disclosed herein. Disclosed are nickase-based gene editing systems designed to effectuate the installation of insertions and / or deletions (indel variants) and / or non-synonymous variants in the coding sequence of LPA. The nickase-based gene editing systems generally comprise one or more mRNAs that encode one or more nickases and a plurality of guide oligonucleotides (e.g., gRNAs) and may be delivered in vivo to a mammalian subject in need thereof via a suitable delivery system, such as lipid nanoparticles (LNPs) (with or without GalNAc targeting moieties) intravenously, or otherwise, administered to a patient as potentially a once-and-done therapeutic. The manufacturing, use, and formulation of the gene editing systems and compositions are also disclosed.

The application belongs to the technical field of medicine, and particularly relates to a liver damage biomarker for judging whether a newborn male piglet is affected by intrauterine growth restriction. The application discloses application of APOA4 as a biomarker for judging whether a male piglet is affected by intrauterine growth restriction. Expression of APOA4 in serum and liver biopsy tissue samples of a male piglet to be detected is determined; when APOA4 is abnormally up-regulated, it is judged that the male piglet to be detected is a male piglet affected by intrauterine growth restriction.

Synthetic nucleic acids are described that can be used for astrocyte-directed expression of heterologous nucleotide sequences, as well as methods of using same for astrocyte-directed expression of such nucleotide sequences for the treatment of neurodegenerative diseases.

The present disclosure relates to engineered phospholipid or lysophospholipid (e.g., sphingosine 1-phosphate (S1P)) chaperones derived from apolipoprotein M (ApoM)-Fc fusion proteins with extended half-life in vivo. The disclosed ApoM-Fc fusion proteins provide a safe, highly efficient and effective way of delivering S1P to endothelial cells and all tissues of the body.

The present disclosure relates to fusion polypeptide comprising a tag moiety and a polypeptide comprising an apolipoprotein or a fragment thereof, for example for. delivery of a loaded decorated liponanoparticle attached via the apoliprotein or a fragment thereof to a cell of interest, wherein said loaded decorated liponanopaticle comprises a payload. Also disclosed are loaded decorated liponanoparticles comprising said fusion polypeptide and a complex comprising a bispecific bindning molecule and the loaded decorated liponanoparticle. Medical uses of the fusion polypeptide, loaded decorated liponanoparticle and complex as disclosed herein are also provided.

Provided herein are engineered fusion proteins comprising ApoM (e.g., human or murine ApoM) fused to a constant region (Fc) of a immunoglobulin G (IgG, e.g., human IgG or murine IgG). In some embodiments, the ApoM-Fc fusion protein further comprises a signal peptide (e.g., IL-2 signal peptide) fused to the ApoM, allowing the fusion protein to be secreted once expressed recombinantly. Methods of using the ApoM-Fc fusion protein or the sponge variants in the treatment of various diseases or disorders are provided.

Provided are a nucleic acid construct for achieving modular loading of an engineered EVs functional protein and use of said construct. A modular design principle is adopted, specific modules are selected for combination, a mutant sequence is optimized and screened to obtain an improved nucleic acid construct, and finally, the engineered EVs modularly loaded with functional protein is converted and expressed. The product has the following advantages that: I) the modular loading of the engineered EVs functional protein is achieved; 2) the loading density and the loading efficiency of the protein inside and outside an EVs membrane are improved; and 3) a specific support sequence module is selected to avoid enzyme digestion. The construct is used to prepare the engineered EVs, so that the modular loading of the functional protein can be achieved, the loading efficiency is improved, and the construct has a wide clinical application value and market prospect.

Compositions and methods for treating rheumatoid arthritis and / or accelerated atherosclerosis are provided, including an inhibitor of oxidized or malondialdehyde-modified low density lipoprotein (LDL) for administration to a subject. Exemplary inhibitors of oxidized LDL include an anti-oxidized LDL antibody, which results in a reduction in the secretion of pro-inflammatory cytokine from primary monocyte in vitro and the plasma cytokine level of inflammatory cytokine in vivo.

Provided herein are compositions and methods for modifying the human gene, APOC3. Such compositions and methods may result in the reduction of the protein, apolipoprotein C3 (apoC-III) when administered to a human subject. Compositions and methods provided herein may comprise a CRISPR-associated (Cas) protein or uses thereof. Compositions and methods of the present disclosure may be useful for treatment of APOC3 associated conditions, including persistent chylomicronemia, familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia (SHTG).

Compositions and methods for treating age-related macular degeneration (AMD) are described herein. In particular, polypeptides comprising a helix structure having ATP-binding cassette transporter membrane stabilization and agonist activity, transporter protein binding activity, and / or capable of effecting cholesterol efflux, and methods of using these peptides for treating AMD are described herein.

This invention relates to antibody-binding lipid nanoparticles comprising antibodies bound to membrane scaffold proteins, and more specifically, to antibody-binding lipid nanoparticles comprising lipids and antibodies bound to membrane scaffold proteins, which are therefore easy to prepare and have excellent specific targeting capabilities.

The present disclosure relates to lipoprotein complexes and lipoprotein populations and their use in the treatment and / or prevention of dyslipidemic diseases, disorders, and / or conditions. The disclosure further relates to recombinant expression of apolipoproteins, purification of apolipoproteins, and production of lipoprotein complexes using thermal cycling-based methods.

A high density lipoprotein particle that does not contain, at least, cholesterol, wherein the high density lipoprotein particle is collected from a culture of hepatocytes; and a method for producing a high density lipoprotein particle that does not contain, at least, cholesterol, the method being characterized in that it comprises culturing hepatocytes, and then collecting the high density lipoprotein particle from the obtained culture.

To provide methods for treating patients with cancer or infection by promoting trained immunity.SOLUTION: Disclosed herein is a nanobiologic composition for promoting trained immunity comprising a nanoscale assembly where the nanoscale assembly is a multi-component carrier composition comprising phospholipids, human apolipoprotein A-I (apoA-I) and NOD2 activator such as a bacterial peptide glycan (e.g., MDP or MTP), where the nanobiologic composition is a nanodisc or nanosphere with a size between about 8 nm and 400 nm in diameter.SELECTED DRAWING: Figure 1

The present disclosure provides a therapeutic nanodisk, the therapeutic nanodisk comprising: a lipid-binding polypeptide; a lipid bilayer and a therapeutic agent, wherein the therapeutic agent may be of use for treating, preventing a central nervous system disease, disorder, trauma or injury; or as a diagnostic agent for diagnosing a central nervous system disease, disorder, trauma or injury. The lipid bilayer may be 1,2-Dimyristoyl-sn-glycero-3-phosphocholine (DMPC) and the therapeutic agent may be a nucleic acid polymer. Further provided are methods for administration of the therapeutic nanodisk to treat, prevent or diagnose the central nervous system disease, disorder, trauma or injury and uses of such therapeutic nanodisks.

Provided is a platform for expressing a protein of interest by artificially manipulating the liver, and more particularly, to a platform for alleviating or treating a genetic disorder or improving a body function by inducing expression by inserting a transgene (e.g., a therapeutic gene) which can function or be expressed normally, into a high-expression secretory gene, instead of a disease gene which functions or is expressed abnormally. The high-expression secretory gene includes the HP or APOC3 gene. The transgene includes one that is highly expressed using a promoter in a hepatocyte genome and is secretory out of the cell.

A gene therapy vector comprising an expression cassette coding for a mammalian apolipoprotein E that has a residue other than arginine at at least one of positions 112, 136, or 158, but is not a mammalian apolipoprotein E that has R112, R136 and R158 or a mammalian apolipoprotein E that has C112, R136 and C158, or coding for an antibody that binds to APOE4 or disrupts the binding of APOE to heparan sulfate proteoglycans, and methods of using the vector, are provided.

To provide oligomeric compounds with conjugate groups targeting apolipoprotein(a).SOLUTION: Provided is an oligomeric compound for use in treating or preventing a disease or condition in a human, the oligomeric compound being ISIS 681257, the treatment comprising administering at most 500 mg of the oligomeric compound to the human during a dosing period.SELECTED DRAWING: Figure 1A