44 results about "Abnormal protein" patented technology

The present invention discloses substituted 2-amino-9-(3,3-difluoropiperidin-4-yl)-7,9-dihydro-8H-purin-8-one compounds compounds having the structural formula represented by formula (I)： The substituted 2-amino-9-(3,3-difluoropiperidin-4-yl)-7,9-dihydro-8H-purin-8-one compounds, derivatives and pharmaceutically acceptable salts thereof of the present invention are capable of acting as a drug characteristic of a protein kinase inhibitor, in particular for the expression of a protein through a DNA-dependent protein kinase (DNA-PK) and can be used to treat or prevent disorders associated with abnormal protein kinase activity, such as cancer, cancer with brain metastases, cancer with meningeal metastases, glioma, glioblastoma, DIPG, and the like either as monotherapy or combination with other treatments, such as radiation therapy or chemotherapy.

Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of PRNP RNA in a cell or animal, and in certain instances reducing the amount of PrP protein in a cell or animal. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease. Such symptoms and hallmarks spongiform changes in the brain, development of abnormal protein aggregates, neuronal loss, markers of neuronal loss, rapidly progressing dementia, and death. Such neurodegenerative diseases include prion diseases, Creutzfeldt-Jakob disease (CJD), variant Creutzfeldt-Jakob Disease (vCJD), familial Creutzfeldt-Jakob Disease (fCJD), Gerstmann-Straussler-Scheinker syndrome, fatal familial insomnia, kuru, Alzheimer's disease, or Parkinson's disease.

The invention relates to methods and compositions for preventing or treating a neuropathology in a subject associated with or induced or caused by a P. gingivalis infection, preventing the deposition of or reducing the level of P. gingivalis gingipain in neuronal tissue, delaying the onset of a P. gingivalis-induced or associated neuropathology, for preventing or slowing the rate of abnormal protein deposition in the neuronal tissue, and / or reducing neuroinflammation, the methods comprising administering an RNA polynucleotide encoding a protein comprising or consisting of: - one or more amino acid sequences of an active site of an Arg- or Lys-gingipain of P. gingivalis, or a sequence that is at least 80% identical thereto; and / or - the amino acid sequence of one or more adhesin binding motifs (ABMs) of an adhesin domain of an Arg- or Lys-gingipain of P. gingivalis, or a sequence that is at least 80% identical thereto, wherein the polynucleotide is capable of being translated in a mammalian cell.

A fusion molecule having phagocytosis-inducing activity is disclosed. The fusion molecule contains a first region capable of binding a TAM receptor and a second region capable of binding to a target substance of which aberrant accumulation is associated with or characteristic of diseases. The fusion molecule effectively clears and / or reduces and / or suppresses accumulated abnormal proteins, such as beta-amyloid, tau, alpha-synuclein, huntingtin, or prion, or the like. Uses of the fusion molecule are disclosed. The fusion molecule can be used for prevention or treatment of proteinosis caused by the abnormal accumulation of substances.

The present invention provides compositions and methods of inhibiting tyrosine phosphorylation. In one aspect, a composition comprising a low-dosage tyrosine kinase inhibitor, where the low-dosage tyrosine kinase inhibitor decreases tyrosine phosphorylation, is provided. In another aspect, a method for treating cardiovascular disease or condition associated with a RASopathy having aberrant protein tyrosine phosphorylation is described. Methods for treating congenital heart disease associated with Noonan or Noonan syndrome with multiple lentigines and decreasing aberrant levels of Protein Zero-Related (PZR) tyrosyl phosphorylation are also described.

The invention relates to the technical field of daily chemicals, in particular to an anti-aging composition and application thereof. The composition is prepared from a white birch bark extract, snake venom peptide and Ectoine. The white birch bark extract promotes abnormal protein removal by activating an intracellular protein ubiquitination system, and improves the skin metabolism capability; the snake venom peptide slows down expression muscle contraction through a nerve regulation mechanism, and dynamic wrinkles are effectively faded; ectoin enhances cell membrane stability, protein protection and skin stress adaptive capacity, and under the synergistic effect of Ectoin, Ectoin and skin stress adaptive capacity, the comprehensive anti-aging effects of resisting wrinkles, tightening, relieving, brightening skin color and the like can be remarkably improved.

To provide a pharmaceutical formulation comprising trehalose for use in treating myopathies, neurodegenerative diseases, or tauopathies associated with abnormal protein aggregation.SOLUTION: A pharmaceutical formulation comprising a therapeutically effective amount of trehalose is provided, wherein the pharmaceutical formulation is for use in a method for treating a disease associated with abnormal protein aggregation and / or inclusion bodies formation in myocytes, neurons, or extracellular compartments, or for alleviating at least one symptom associated therewith in a human subject in need thereof, the method comprising intravenously administering to the subject a pharmaceutical formulation comprising 0.1% (w / v) to 50% (w / v) of trehalose. An injectable aqueous pharmaceutical formulation comprising a therapeutically effective amount of trehalose is also provided.SELECTED DRAWING: None

The purpose of the present invention is to suppress the effect of ultraviolet light on melanocytes. Upon performance of gene expression analysis with a focus on the olfactory receptor OR2L13 expressed by melanocytes, it was discovered that addition of (−)-carvone, a ligand of OR2L13, increased the expression of genes related to suppression of active oxygen, suppression of the production of inflammatory cytokines or chemokines, suppression of senescence, and / or degradation of abnormal proteins. As a result of said discovery, the present invention provides an inhibitor of the effects of ultraviolet light on melanocytes that includes (−)-carvone.

The invention belongs to the technical field of medical equipment, and particularly relates to an intracranial effusion drainage device which comprises a bottom plate, a drainage suction adjusting mechanism, an intracranial effusion storage mechanism, a drainage height adjusting mechanism and an automatic drainage anti-blocking mechanism. The drainage suction adjusting mechanism, the intracranial effusion storage mechanism and the drainage height adjusting mechanism are all arranged at the top of the bottom plate, and the drainage automatic anti-blocking mechanism is arranged on the drainage height adjusting mechanism; the problems that when abnormal protein tissue exists in intracranial hydrops of a patient, an existing drainage device is prone to being blocked, and suction during drainage cannot be adjusted through the existing drainage device are solved.

Disclosed is a method of treatment of a disease associated with abnormal protein aggregation comprising parenterally administering pharmaceutical formulations comprising trehalose. Also disclosed is an injectable aqueous pharmaceutical formulation comprising a therapeutically effective amount of trehalose.

The present invention relates to series of peptidomimetic compounds as an inhibitor targeting protein N-terminal methyltransferase pharmacological pathway. Pharmaceutical compositions of those compounds and methods of using them in the treatment of diseases caused by abnormal protein methyltransferase pathway, including cancer, inflammation, neurodegenerative and cardiovascular diseases, are within the scope of this disclosure.

The present application relates to TAT-PO1 and its application in treating amyotrophic lateral sclerosis (ALS). The polypeptide connects TAT sequence (SEQ ID NO. 1) and PO1 sequence (SEQ ID NO. 2) through amino hexanoic acid (AHX), and its amino acid sequence is YGRKKRRQRRR{AHX}RHIFLIRHSQYHVDGSLEKDRTLTPLGREQAE. TAT-PO1 can competitively block the interaction between PGAM5 and OMA1, thereby interfering with the pathological mechanism of ALS. The polypeptide TAT-PO1 of the present application can be prepared into a drug, and directly targets the central nervous system through intrathecal injection or intravenous administration, thereby providing a new strategy for the treatment of ALS. In addition, the design idea can also be applied to other neurodegenerative diseases caused by abnormal protein interaction, thereby providing a technical paradigm for the development of related drugs.

Diagnostic and therapeutic methods for neurodegenerative diseases. Are provided involving assaying abnormal proteins (hyperphosphorylated tau, α-synuclein, TDP-43) associated with neuronal turnover inhibition or promotion in patient samples. Abnormal protein expression and apoptotic activity are detected, aiding disease progression assessment. Therapeutically, a method is provided for treating neurodegenerative diseases, administering compounds promoting neuronal turnover or modulating proteins involved in the process. The invention extends to identifying suitable drugs, employing neuronal turnover induction, miRNA modulation, and protein activity inhibition or enhancement. The claims also encompass various species, tissues, and cultured cells. Furthermore, the invention is applicable to diverse neurodegenerative diseases with abnormal protein accumulation, presenting novel diagnostic and treatment approaches.

The present invention relates to series of peptidomimetic compounds as an inhibitor targeting protein N-terminal methyltransferase pharmacological pathway. Pharmaceutical compositions of those compounds and methods of using them in the treatment of diseases caused by abnormal protein methyltransferase pathway, including cancer, inflammation, neurodegenerative and cardiovascular diseases, are within the scope of this disclosure.

The invention relates to a magnetic activation preparation method and application of phagocytic anti-inflammatory phenotype microglial cells, and the method comprises the following steps: separating and culturing microglial cells, and after the cells are cultured to a preset fusion degree, stimulating the microglial cells by using a pulsed magnetic field to obtain high-phagocytic and high-anti-inflammatory phenotype microglial cells which are transplanted into a specific brain region, the pulse magnetic field is used for treating central nervous cell diseases, the stimulation frequency of the pulse magnetic field is 1-100 Hz, the number of stimulation pulses is larger than or equal to 500, and the number of stimulation days is larger than or equal to 1 day. The microglial cells obtained by adopting low-frequency pulse stimulation have stronger migration ability, can swallow more foreign matters or abnormal protein aggregation products, can secrete less proinflammatory factors, is beneficial to recovery of the microenvironment of the central nervous system, and can be used for improving the microenvironment of the central nervous system. The invention provides an efficient, accurate and economical new way for microglial cells to treat neuroinflammation regulation and control and abnormal protein aggregation related diseases, and solves the technical problems of large side effect, high cost, inaccurate target spot, difficulty in acting on deep microglial cells and the like in the prior art.

The present invention relates to series of peptidomimetic compounds as an inhibitor targeting protein N-terminal methyltransferase pharmacological pathway. Pharmaceutical compositions of those compounds and methods of using them in the treatment of diseases caused by abnormal protein methyltransferase pathway, including cancer, inflammation, neurodegenerative and cardiovascular diseases, are within the scope of this disclosure.

To provide a method of treating a paraproteinemic neuropathy in a subject by administering to the subject an antagonist of the complement system.SOLUTION: An antagonist of the complement system inhibits the complement system upstream of complement factor C5. More specifically, the antagonist may be an antibody or an antigen-binding fragment thereof that blocks or inhibits the complement system by inhibiting the C2b domain of complement factor C2. The paraproteinemic neuropathies that may be treated include, in particular, multifocal motor neuropathy (MMN), chronic inflammatory demyelinating polyneuropathy (CIDP), and Guillain-Barre syndrome (GBS).SELECTED DRAWING: Figure 1

The present invention relates to a drug for treating or preventing a central nervous system disease whose cause is an abnormal aggregation of proteins in the brain, comprising, as an active ingredient, a compound of formula (1) (wherein R1 and R2 are hydrogen or the like, R3 and R4 are hydrogen, C1-6 alkyl or the like, R5 is halogen, C1-6 alkyl or the like, R6 is hydrogen, halogen or the like, X is oxygen or the like, Y is carbon or the like, and a pharmaceutically acceptable salt thereof, in the formula, m and n are integers of 0, 1 or the like, r and s are 0, 1, 2 or the like, and Hy is a pyridine ring or the like, which has an effect of inhibiting or reducing the accumulation of abnormal proteins in the brain. # imgabs0 #

Provided herein are methods for treating conditions and diseases characterized by SCN1A, SCN8A, or SCN5A protein deficiency by targeting variable splicing events in the SCN1A gene and modulating expression levels of functional proteins and / or inhibiting aberrant protein expression in Delavir Syndrome patients.

Compounds that can promote the expression of SCN1A, a specific gene, are provided herein. In some embodiments, compositions, methods, and kits related to the compounds disclosed herein are provided herein. In some embodiments, the compounds provided herein can target alternative splicing events in the SCN1A gene, can regulate the expression level of functional proteins in patients with Dravet syndrome, and / or can inhibit abnormal protein expression. Such compounds can be used to treat conditions caused by deficiencies in SCN1A protein, SCN8A protein, or SCN5A protein.

The present invention relates to a drug for treating or preventing central nervous system diseases caused by abnormal aggregation of proteins in the brain, which comprises, as an active ingredient, a compound represented by formula (1) (wherein R1 and R2 are hydrogen or the like, R3 and R4 are hydrogen, C1-6 alkyl or the like, X is oxygen or the like, Y is carbon or the like, Z is C1-6 alkyl or the like, and m and n are integers of 0, 1, 2 or the like) or a pharmaceutically acceptable salt thereof. The compound has the effect of inhibiting or reducing abnormal protein aggregate accumulation in the brain. # imgabs0 #

The invention relates to TAT-PO1 and application of TAT-PO1 in treatment of neurodegenerative disease amyotrophic lateral sclerosis. The polypeptide is connected with a TAT sequence (SEQ ID NO.1) and a PO1 sequence (SEQ ID NO.2) through aminocaproic acid (AHX), and the amino acid sequence of the polypeptide is as follows: YGRKKRRQRRR {AHX} RHIFLIRHSQYHVDGSLEKDRTLTPLGREQAE. The TAT-PO1 can competitively block the interaction between the PGAM5 and the OMA1, so that the pathological mechanism of the ALS is intervened. The polypeptide TAT-PO1 disclosed by the invention can be prepared into a medicine, and directly targets a central nervous system through intrathecal injection or intravenous administration, so that a new strategy is provided for ALS treatment. In addition, the design thought can also be applied to other neurodegenerative diseases caused by abnormal protein interaction, and a technical normal form is provided for development of related drugs.

The present invention discloses a protein molecular marker C3 for non-invasive prenatal diagnosis of non-syndromic cleft lip and palate fetuses and its application, belonging to the field of pharmaceutical biotechnology. The present invention utilizes proteomics detection platform technology and ELISA experimental methods to compare differentially expressed proteins in the serum of pregnant women with non-syndromic cleft lip and palate fetuses and pregnant women with healthy fetuses, and then analyzes the correlation between the differentially expressed proteins and the disease. Based on a large number of specimens from the training cohort and the validation cohort, it was discovered and confirmed for the first time that abnormal expression of protein C3 in the serum of pregnant women is closely correlated with the occurrence of non-syndromic cleft lip and palate fetuses, and can be used alone for prenatal screening of non-syndromic cleft lip and palate, providing a new target for the treatment of non-syndromic cleft lip and palate, which has great clinical significance.

A fusion molecule having phagocytosis-inducing activity is disclosed. The fusion molecule contains a first region capable of binding a TAM receptor and a second region capable of binding to a target substance of which aberrant accumulation is associated with or characteristic of diseases. The fusion molecule effectively clears and / or reduces and / or suppresses accumulated abnormal proteins, such as beta-amyloid, tau, alpha-synuclein, huntingtin, or prion, or the like. Uses of the fusion molecule are disclosed. The fusion molecule can be used for prevention or treatment of proteinosis caused by the abnormal accumulation of substances.

The present invention provides compositions and methods of inhibiting tyrosine phosphorylation. In one aspect, a composition includes comprising a low-dosage tyrosine kinase inhibitor, where the low-dosage tyrosine kinase inhibitor decreases tyrosine phosphorylation. In another aspect, a method is described for treating cardiovascular disease or condition associated with a RASopathy having aberrant protein tyrosine phosphorylation. Methods for treating congenital heart disease associated with Noonan or Noonan syndrome with multiple lentigines and decreasing aberrant levels of Protein Zero-Related (PZR) tyrosyl phosphorylation are also described.

Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of PRNP RNA in a cell or animal, and in certain instances reducing the amount of PrP protein in a cell or animal. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease. Such symptoms and hallmarks spongiform changes in the brain, development of abnormal protein aggregates, neuronal loss, markers of neuronal loss, rapidly progressing dementia, and death. Such neurodegenerative diseases include prion diseases, Creutzfeldt-Jakob disease (CJD), variant Creutzfeldt-Jakob Disease (vCJD), familial Creutzfeldt-Jakob Disease (fCJD), Gerstmann-Straussler-Scheinker syndrome, fatal familial insomnia, kuru, Alzheimer's disease, or Parkinson's disease.

The invention discloses application of RPS29 protein in preparation of products or medicines for diagnosing and treating amyotrophic lateral sclerosis, and belongs to the technical field of biological medicines. The reagent for further detecting the expression level of the RPS29 protein is used for preparing a product for diagnosing amyotrophic lateral sclerosis, such as a kit for detecting the expression level of the RPS29 protein; or the RPS29 protein expression up-regulating agent is used for preparing a medicine for treating amyotrophic lateral sclerosis, such as a targeted medicine taking RPS29 protein as a target spot, so that the problems that in the prior art, stable and sensitive ALS early molecular diagnosis markers are lacked, disease recognition is delayed, intervention cannot be carried out before neuronal functions are seriously damaged, and the treatment effect is poor are solved. And a targeted treatment means for protein translation abnormality and neuronal survival disorder in ALS is lacked, meanwhile, the urgent requirements for ALS early screening and targeted treatment are met, and the clinical potential is high.

The present disclosure provides methods for preventing or treating a neurodegenerative disorder in a subject, where the neurodegenerative disorder is one characterized by a deposition of abnormal tau protein in the brain of the subject. The method generally includes administering to the subject a therapeutically effective amount of a drug that is a tau propagation antagonist.

This invention belongs to the field of biotechnology, specifically relating to a monoclonal antibody FHY-001 for the treatment of nasopharyngeal carcinoma and its applications. The monoclonal antibody FHY-001 comprises heavy chain variable regions of VHCDR1, VHCDR2, and VHCDR3, and light chain variable regions comprising VLCDR1, VLCDR2, and VLCDR1. This invention focuses on the research and development of monoclonal antibodies targeting specific and effective therapeutic targets for nasopharyngeal carcinoma. Specifically, it utilizes abnormal proteins encoded by mutated genes in nasopharyngeal carcinoma tumor cells, which can be recognized by immune cells and activate the body's immune response, or neoantigens derived from non-human normal genomes, to prepare specific novel antibodies for the treatment of nasopharyngeal carcinoma. The monoclonal antibody of this invention exhibits high specificity, specifically binding to human nasopharyngeal carcinoma tumor cells, and significantly inhibits the growth of nasopharyngeal carcinoma cells in vitro. It also has a high target binding rate, can be mass-produced, and can serve as a potential drug for the immunotherapy of nasopharyngeal carcinoma.

The invention provides a preserved egg appearance grade classification system and method based on machine vision, machine vision identifies appearance characteristics of preserved egg shells, determines structure abnormal areas and color abnormal areas of the preserved egg shells, and comprehensively identifies condition problems possibly caused in a preserved egg pickling process; according to the structure abnormal region, estimating the penetration state characteristics of the pickling ingredients in the preserved egg so as to calibrate the solidification abnormal region in the preserved egg; according to the color abnormal area, a metal salt accumulation area in the preserved egg is calibrated, and abnormal protein solidification and excessive metal element enrichment which possibly occur in the preserved egg in the pickling process are estimated through the appearance state of the preserved egg; the preserved eggs are graded and marked according to the abnormal solidification area and the metal salt accumulation area, the preserved eggs are subjected to appearance visual recognition to determine the pickling quality of the preserved eggs, the preserved eggs are quickly and accurately recognized and screened, and the production efficiency and quality of the preserved eggs are improved.