4149results about "Genetic material ingredients" patented technology

The invention provides a production strain of an antibiotic-free plasmid, the production strain is a gene editing strain of a PIR strain and is named as PIR1-WN:: 0636 or PIR1-PR: 0636, the production strain contains a nucleotide sequence for coding toxin protein and the antibiotic-free plasmid, and the antibiotic-free plasmid contains a nucleotide sequence for coding antitoxin protein; and preferably, the replicon DNA element of the nonreactive plasmid is R6K-gamma. The toxin protein gene of the production strain disclosed by the invention can be stably passaged, has lethality after being induced and can be used for plasmid screening; according to the invention, the positive rate of transforming the nonreactive plasmid into the PIR1-WN:: 0636 strain is more than 80%, and stable production of the plasmid with a high superhelix ratio can be realized.

The invention discloses an engineered bionic nucleic acid nano-vesicle as well as a preparation method and application thereof, relates to the technical field of nano biomedicine, and aims at solving the problems that in-vivo targeting efficiency and immunogenicity of a traditional cationic lipid nano-carrier are limited due to deletion and cleavage obstacles of GSDMD expression in tumor cells. The technical key point of the invention is as follows: the engineered bionic nucleic acid nano-vesicle is provided and is prepared by wrapping a cationic lipid nucleic acid drug with an exosome derived from engineered macrophages; wherein the exosome from the engineered macrophage is the exosome from the macrophage with high expression of PD1, which is as shown in SEQ. ID. NO.1. The exosome from the engineered macrophage is the exosome from the macrophage with high expression of PD1; the lipid nucleic acid medicine is prepared by loading GSDMD-N mRNA (messenger Ribonucleic Acid) shown on the basis of SEQ.ID.NO.2 on a cationic liposome. The engineered bionic nucleic acid nano-vesicle is used for preparing an oral squamous cell carcinoma diagnostic kit and a therapeutic drug.

Described herein are muscle-specific targeting moieties and compositions including the muscle specific targeting motifs. Also described herein are uses of the muscle-specific targeting motifs and compositions including the muscle specific targeting moieties. In some embodiments, the muscle-specific targeting moieties and compositions including the muscle specific targeting moieties can be used to direct delivery of a cargo to a muscle cell.

Described herein are targeting moieties that can be capable of specifically targeting muscle cells and can include an n-mer motif. In some embodiments, the n-mer motif contains an RGD motif. Also described herein are vector systems, particles, polypeptides that can encode and / or contain one or more targeting moieties. Also described herein are methods of delivering a cargo to a cell, such as a muscle cell, using one or more of the targeting moieties described herein.

The present invention relates to a novel lipid compound for tissue-specific delivery, and a lipid nano-particle (LNP) composition comprising the same, the lipid nano-particle comprising a modified lipid compound as a component, according to the present invention, internal active substances are selectively delivered into cells of specific tissues such as lymph nodes, spleen, retina, cancer, brain, liver and the like in vivo, thereby preventing side effects and safely exhibiting a desired level of effect. The tissue-specific non-viral LNP delivery vectors can be effectively used for prevention of infectious diseases and treatment of rare and refractory (hereditary) diseases (diseases which are effectively and selectively delivered to in-vivo targeted sites, such as macular degeneration, diabetic retinal degeneration, hereditary retinal degeneration, cancer, cerebral diseases, liver diseases and the like).

Aspects of the present disclosure relate to conjugates comprising a muscle targeting agent covalently linked to a molecular payload. In some embodiments, the muscle targeting agent specifically binds to an internalized cell surface receptor on muscle cells. In some embodiments, the molecular payload promotes the expression or activity of functional dystrophin protein. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide, e.g., an oligonucleotide that causes exon skipping in mRNA expressed from a mutant DMD allele.

The invention relates to a gene, a protein and a plasmid of a trachinotus ovatus apoptosis regulatory factor Bok and application of the gene, the protein and the plasmid, and belongs to the field of molecular biology, the sequence of cDNA nucleotide of the trachinotus ovatus apoptosis regulatory factor Bok is as shown in SEQ ID NO.1, and the amino acid sequence is as shown in SEQ ID NO.2. The invention further provides a construction method of the eukaryotic expression plasmid of the trachinotus ovatus apoptosis regulatory factor Bok. The regulation effect of the constructed eukaryotic expression vector on other apoptosis-related factors of the trachinotus ovatus is verified, and after the eukaryotic expression plasmid for the apoptosis regulation factor Bok of the trachinotus ovatus is injected into a fish body, the disease resistance of the fish body can be remarkably improved, and a remarkable immune protection effect is achieved.

The present invention provides a method for producing nucleic acid-encapsulating lipid nanoparticles, including the following steps (a) and (b): step (a) of mixing an alcohol solution containing an ionic lipid having a tertiary amino group, a sterol, and a PEG lipid with a citrate buffer having pH 3 to 6.5 in which nucleic acid is dispersed to prepare a suspension of nucleic acid-encapsulating lipid nanoparticles; and step (b) of exchanging a dispersion medium of the aforementioned suspension for a Tris buffer having pH 5.2 to 9.0 by concentrating the suspension of nucleic acid-encapsulating lipid nanoparticles by ultrafiltration and diluting same with the aforementioned Tris buffer.

The invention discloses application of a JMJD6-targeted reagent in preparation of a medicine for promoting myocardial cell proliferation. The medicine can promote myocardial cell proliferation after myocardial infarction and reduce the myocardial fibrosis scar area caused by myocardial infarction. AAV9 myocardial specific overexpression virus and JMJD6 myocardial specific knockout mice are utilized, and the positive effect of JMJD6 in promotion of P1 cardiac apex resection of newborn mice and regeneration and repair of injured hearts after myocardial infarction of adult mice is disclosed for the first time; the specific mechanism is that JMJD6 depends on the activity of histone demethylase, enrichment of active modification H4R3me2a and H3R2me2s in a PDK4 promoter region is removed, transcriptional expression of the H4R3me2a and the H3R2me2s is inhibited, an impaired heart energy substrate utilization mode is stimulated to be increased and converted from fatty acid oxidation energy supply to glycolysis oxidation energy supply, and then adult myocardial cell proliferation is effectively promoted. The regeneration and repair capability of the heart after myocardial infarction is greatly improved, and a new effective target spot is provided for clinical treatment of myocardial injury.

The invention discloses a recombinant adeno-associated virus vector for targeted delivery of VEGF-C (vascular endothelial growth factor-C) and application of the recombinant adeno-associated virus vector in promoting endocardial differentiation to treat coronary heart disease. The rAAV vector comprises transgene for coding VEGF-C protein or VEGF-C protein functional variant and a tissue-specific promoter operably connected to the transgene, wherein the tissue specific promoter is capable of driving the transgene to be specifically expressed in the heart endothelial cells. According to the treatment method developed by the invention, specific expression of the Vegfc gene in the heart is driven by using an ICAM2 promoter with targeting to endothelial cells, and a remarkable curative effect is shown in a myocardial infarction model mouse. The therapeutic scheme provides a new effective strategy for intervention and treatment of myocardial infarction and chronic heart failure, and has the advantages of high safety, long gene expression duration, extremely low immune response to a host and the like.

Lipid nanoparticle compositions for the delivery of nucleic acids are provided.SOLUTION: In various embodiments, the lipid nanoparticle comprises an ionizable lipid of Formula (I). Also provided are methods of using such lipid nanoparticle compositions to achieve targeted delivery of therapeutic cargo without the need for a targeting ligand.SELECTED DRAWING: None

The purpose of the present invention is to provide: lipid nanoparticles useful as a nucleic acid delivery carrier; and a novel pH-sensitive cationic lipid for producing the lipid nanoparticles. The problem is solved by: a pH-sensitive cationic lipid comprising an asymmetric hydrocarbon chain; and lipid nanoparticles comprising the pH-sensitive cationic lipid as a constituent lipid.

The present invention relates to a compound represented by formula (I) or a stereoisomer, tautomer, solvate or pharmaceutically acceptable salt of a compound represented by formula (I), TIFF2026503201000033.tif3981X1, X2 and X3 are each independently an optionally substituted C1-C 15 alkylene, and R and R are each independently an optionally substituted C-C 40 Alkyl, optionally substituted C-C 40 Heteroalkyl, optionally substituted C-C 40 Alkenyl, optionally substituted C-C 40 Heteroalkenyl, optionally substituted C-C 40 Alkynyl or optionally substituted C-C 40 The present invention provides a compound comprising heteroalkynyl, wherein R3, R4, R5, and R6 are each independently H, halogen, or optionally substituted C1-C3 alkyl, and the substituents are independently selected from halogen, -OH, -SH, -NH2, -NO2, cyano, and C1-C3 alkyl, which has the advantages of low cytotoxicity, strong delivery ability, and strong immunostimulatory effect.

Compounds and compositions capable of modulating immune responses in patients, as well as implantable elements containing them, are provided. [Solution] For example, 4-((1-(2-(2-(2-(2-aminoethoxy)ethoxy)ethoxy)ethyl)-1H-1,2,3-triazol-4-yl)methyl)thiomorpholine 1,1-dioxide, as well as pharmaceutically acceptable salts, solvates, hydrates, tautomers, stereoisomers, isotopically labeled derivatives and compositions thereof, and implantable elements (e.g., devices and materials) containing these are provided.

The present invention relates to the field of non-viral vectors and pharmaceutical compositions comprising polypropyleneimine and nucleic acids, and their use in human or veterinary medicine. More particularly, the present invention relates to pharmaceutical compositions comprising polypropyleneimine polymers or copolymers for the delivery or transfection of nucleic acids, such as RNA. The pharmaceutical compositions described herein are particularly useful for (nucleic acid) vaccination, nucleic acid-based protein therapy, nucleic acid-based protein replacement therapy, gene editing, base editing, cell therapy, immunotherapy, stem cell therapy, regenerative medicine, gene silencing, nucleic acid inhibition or protein inhibition.

The invention discloses an FAM20B protein and application of an FAM20B protein inhibitor in prevention or treatment of glucolipid metabolism related diseases. Specifically, the invention discloses an application of an FAM20B protein and / or an FAM20B gene in regulation and control of liver glycolipid metabolism. The invention also discloses application of the FAM20B inhibitor in improving, preventing or treating liver glycolipid metabolism disorder or glycolipid metabolism related diseases (such as diabetes, metabolism related fatty liver disease MAFLD, obesity and the like). Experiments show that the FAM20B inhibitor can reduce the liver gluconeogenesis level, relieve impairment of glucose tolerance, relieve hyperglycemia of diabetic patients, reduce the fat content of the whole body and the liver, relieve liver lipid deposition, promote energy consumption, reduce food intake, promote fat burning and reduce liver fat accumulation; the compound can be used for preparing medicines for preventing and treating glucose and lipid metabolism disorder or glucose and lipid metabolism related diseases, and has a wide application prospect in the fields of prevention and treatment of metabolism related diseases.

The present invention provides a bacteriophage having a bacteriolytic activity against Mycobacterium avium and / or Mycobacterium intracellularis, the bacteriophage having a genome containing a nucleic acid sequence represented by the genome of the bacteriophage specified by the preservation number NITE BP-03513 or NITE BP-03514 or the preservation number NITE BP-03918, the bacteriophage having a bacteriolytic activity against Mycobacterium avium and / or Mycobacterium intracellularis, and the bacteriophage having a bacteriolytic activity against Mycobacterium avium and / or Mycobacterium intracellularis. The capsid of the phage is connected with the cell-penetrating peptide through a tag.

The present invention relates to a saponin-based saponin derivative comprising a triterpene aglycone and a first and / or second sugar chain, the saponin derivative comprising: an aglycone core structure containing a derivatized aldehyde group; and / or the first sugar chain containing a derivatized carboxyl group; and / or the second sugar chain containing at least one derivatized acetoxy group. The present invention also relates to a first pharmaceutical composition comprising the saponin derivative of the present invention. In addition, the present invention relates to a pharmaceutical combination comprising the first pharmaceutical composition of the present invention and a second pharmaceutical composition comprising any one or more of an antibody-toxin conjugate, a receptor-ligand-toxin conjugate, an antibody-drug conjugate, a receptor-ligand-drug conjugate, an antibody-oligonucleotide conjugate, or a receptor-ligand-oligonucleotide conjugate. The present invention also relates to the first pharmaceutical composition or the pharmaceutical combination of the present invention for use as a medicament or in the treatment or prevention of cancer, an infectious disease, a viral infection, hypercholesterolemia, primary hyperoxaluria, hemophilia A, hemophilia B, alpha-1 antitrypsin-associated liver disease, acute hepatic porphyria, transthyretin-mediated amyloidosis, or an autoimmune disease. Furthermore, the present invention relates to an in vitro or ex vivo method for translocating a molecule from outside a cell into the cell, which method comprises contacting the cell with the molecule and with a saponin derivative of the present invention.

A CVB virus expression vector comprising a PIV5 W3A viral genome comprising a mutation at amino acid residue S157 or S156 of the P / V gene and a deletion of the small hydrophobic (SH) gene of the PIV5 W3A viral genome, wherein the amino acid substitution at amino acid residue S157 or S156 comprises a substitution of serine (S) with phenylalanine (F) or asparagine (N), and the SH gene has a deletion of the SH open reading frame or the entire SH gene transcription unit. The CVB virus expression vector expresses a heterologous polypeptide, including SARS-CoV-2 spike (S), and / or nucleocapsid (N) and / or membrane (M) proteins, RSV fusion protein (F), or other antigens.

This disclosure relates to compositions and methods using genetically modified bacteria that express superoxide dismutase (SOD) and catalase enzymes to reduce oxidative stress in the skin, and can be used for various skin conditions. In some embodiments of this disclosure, the genetically modified bacteria are Gram-positive bacteria, for example, of the genus Corynebacterium.

New synthetic expression cassettes comprising a minimal promoter and an immune cell- specific enhancer for expression of a nucleic acid of interest in immune cells such as T cells and NK cells are disclosed. Vectors and host cells comprising such synthetic expression cassettes are also disclosed. The application also discloses methods for expressing a nucleic acid of interest, such as a nucleic acid encoding a chimeric antigen receptor (CAR), in a cell and for treating diseases or conditions such as cancers and genetic diseases using the synthetic expression cassettes, vectors and cells.

This invention provides a novel compound or a pharmacologically acceptable salt thereof that inhibits the expression of the CALM2 gene, which is known to be involved in congenital LQTS. [Solution] The present invention provides a compound or a pharmacologically acceptable salt thereof comprising a modified oligonucleotide having a length of 8 to 80 consecutive nucleosides, wherein the modified oligonucleotide has a nucleic acid base sequence comprising at least 8 consecutive nucleic acid bases included in a specific sequence. The compound or a pharmacologically acceptable salt thereof makes it possible to treat diseases or conditions (particularly congenital long QT syndrome) in which inhibition of CALM2 gene expression by controlling CALM2 gene expression is effective.

The present disclosure provides a construct comprising a coding sequence operably linked to a promoter, wherein the coding sequence encodes a vascular endothelial growth factor (VEGF) binding agent or a portion thereof. In some embodiments, a construct is an AAV construct. In some embodiments, an AAV construct is a part of an AAV particle. Compositions comprising constructs and AAV particles described herein can be useful in treating hearing loss, for example, hearing loss associated with vestibular schwannoma.

The present invention provides a pharmaceutical composition for treating cancer, the pharmaceutical composition comprising a nucleic acid that suppresses the expression of Chromosome 11 Open Reading Frame 97 (C11orf97). With the present invention, it becomes possible to treat cancer.

The present invention provides an hBCMA-binding polypeptide comprising at least one motif that binds to hBCMA, wherein the peptide has the following structure: [N-terminal portion]-[Helix 1]-[Spacer portion]-[Helix 2]-[C-terminal portion], and the hBCMA-binding motif is the portion [Helix 1]-[Spacer portion]-[Helix 2]. The present invention further provides a pharmaceutical composition comprising the hBCMA-binding polypeptide, and the use of the hBCMA-binding polypeptide or the pharmaceutical composition for use as a medicament, particularly for the treatment or prevention of cancer.

This specification provides a mutant adeno-associated virus serotype 9 (AAV9) capsid protein and recombinant AAV9 particles containing the mutant AAV9 capsid protein. Further provided are a pharmaceutical composition comprising recombinant AAV9 particles, a polynucleotide encoding the mutant AAV9 capsid protein, a vector and host cells containing the polynucleotide, a population of host cells transduced by the recombinant AAV9 particles, and various methods for using the recombinant AAV9 particles.

The invention relates to a pharmaceutical composition containing a nucleic acid construct and a medical application thereof. Specifically, the invention relates to lipid nanoparticles as shown in a formula I, which can realize efficient delivery of exogenous target genes, so that the exogenous target genes are efficiently and quickly expressed in vivo, have the advantages of no gene integration risk and easiness in industrial amplification, are more ideal therapeutic schemes than naked plasmids, and can be used as gene therapeutic drugs for various diseases.