13 results about "Aberrant protein" patented technology

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.

The present invention provides a polypeptide that specifically recognizes structurally abnormal proteins generated in mammals due to misfolding or the like, and an agent for detecting or reducing structurally abnormal proteins that uses the polypeptide. The present invention relates to an agent for detecting structurally abnormal proteins, comprising as an active ingredient a polypeptide having structurally abnormal proteins-binding site which is a polypeptide consisting of the amino acid sequence represented by SEQ ID NO:2, or a polypeptide consisting of an amino acid sequence having 90% or more sequence identity with the amino acid sequence and having binding activity to structurally abnormal proteins, and an agent for reducing structurally abnormal proteins comprising as an active ingredient a polypeptide containing the structurally abnormal protein binding site and a ubiquitin ligase active site.

A variable splicing event in a gene can result in a non-productive mRNA transcript, which in turn can result in abnormal protein expression, and a therapeutic agent that can target a variable splicing event in a gene can modulate the expression level of a functional protein and / or inhibit abnormal protein expression in a patient. Such therapeutic agents are useful for the treatment of conditions or diseases caused by protein deficiency and / or mitochondrial function deficiency.

Alternative splicing events in genes can lead to non-productive mRNA transcripts which in turn can lead to aberrant or reduced protein expression, and therapeutic agents which can target the alternative splicing events in the genes can modulate the expression level of functional proteins in patients and / or inhibit aberrant protein expression. Such therapeutic agents can be used to treat a condition or disease caused by protein deficiency.

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.

Described herein are polypeptide useful for identifying compound for modulating aberrant protein condensates. The polypeptide comprise a condensate-modulating protein, a first biomarker polypeptide, a barcoding biomarker polypeptide. When inside a cell, the non-natural polypeptide exist as multiple fragments, including a first fragment comprising the condensate-modulating protein and the first biomarker polypeptide, and a second fragment comprising the barcoding biomarker polypeptide. The barcoding biomarker polypeptide is uniquely associated with and identifies the cell expressing associated condensate-modulating protein. Also described is a polynucleotide encoding the non-natural polypeptide, a library of the polynucleotide, and a method of identifying compound for modulating aberrant protein condensates using the non-natural polypeptide, the construct, and / or the library.

Alternative splicing events in genes can lead to non-productive mRNA transcripts which in turn can affect protein expression level, and therapeutic agents which can target the alternative splicing events in genes can modulate the expression level of functional proteins in patients and / or inhibit aberrant protein expression. Such therapeutic agents can be used to treat a condition or disease caused by protein deficiency.

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 is an agent for inhibiting abnormal protein aggregate accumulation or an agent for inhibiting the uptake of an abnormal protein into a neuron, particularly an agent for preventing or treating a disease associated with accumulation of abnormal protein aggregates, in which the agent contains an RGMa inhibiting substance.

The invention belongs to the technical field of biology, and particularly relates to a composition for improving the developmental potential of oocytes, an in-vitro maturation culture solution and an optimized culture method. And a CCR5 / CXCR3 antagonist. The preparation for in-vitro embryo production contains an Ephrin ligand family and a CCR5 / CXCR3 antagonist, the microenvironment for maturation culture of oocytes is synergistically optimized, further, sesquiterpenol compounds and / or phenylpropane compounds can be added on the basis, and therefore the preparation can be used for preparing the embryos in vitro by means of the synergistic effect of the components. The composition can effectively inhibit the formation of abnormal protein aggregates in oocytes, enhance the ability of proteasomes to remove error protein aggregates, significantly reduce the level of reactive oxygen species (ROS) and increase the content of endogenous antioxidant substances such as glutathione (GSH), so as to reduce the protein toxicity stress, oxidative stress and other dimensions, and thus, the composition can be used for preparing an anti-inflammatory drug. The quality and the fertilization rate of oocytes after in-vitro maturation and the development potential of subsequent embryos are remarkably improved, and the application prospect is wide.

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.

Disclosed are compounds and pharmaceutically acceptable salts and stereoisomers thereof that may cause degradation of various proteins e.g., IKZF2 (Helios). Also disclosed are pharmaceutical compositions containing same, and methods of making and using the compounds to treat diseases and disorders characterized or mediated by aberrant protein activity.

Alternative splicing events in SCN1A gene can lead to non-productive mRNA transcripts which in turn can lead to aberrant protein expression, and therapeutic agents which can target the alternative splicing events in SCN1A gene can modulate the expression level of functional proteins in Dravet Syndrome patients and / or inhibit aberrant protein expression. Such therapeutic agents can be used to treat a condition caused by SCN1A, SCN8A or SCN5A protein deficiency.