33 results about "Cell type specific" patented technology

The present invention provides targeting polyplexes comprised of (i) nucleic acids, in particular nucleic acids encoding pharmaceutically active peptides or proteins, and (ii) targeting conjugates comprising LPEI and PEG fragments that are connected by discrete linkages formed through defined, chemoselective reactions. Thus, the LPEI fragment is bonded in a linear end- to-end fashion to a single PEG fragment. The linear conjugates are further conjugated to a targeting fragment to enable selective interaction with a particular cell type. The polyplexes selectively deliver the nucleic acids to the targeted cells resulting in high expression and efficient protein translation as well as secretion of the encoded pharmaceutically active proteins.

Provided is a viral vector having a lipid bilayer envelope, the lipid bilayer envelope comprising an antibody binding domain displayed outside the envelope, the antibody binding domain being cell type specific; a viral envelope protein exhibited outside the envelope, the viral envelope protein being capable of promoting infection of the same cell type; and a nucleic acid molecule comprising a promoter capable of being expressed in the same cell type. Methods of making the viral vectors and methods of modifying cells and treating diseases / conditions using the viral vectors are also provided.

The invention discloses a method for constructing a neurodevelopmental disorder animal model based on central nervous system myelin sheath function change and application, and belongs to the technical field of biological engineering. An Msl2 gene conditional knockout mouse model is constructed by adopting a gene engineering technology, the space-time specific knockout of a second exon of the Msl2 gene in a specific cell type is realized through a Cre-LoxP recombinase system, and the exon encodes a key enzyme activity region for catalyzing ubiquitination. Model construction is based on central nervous system oligodendrocyte / myelin sheath dysfunction, the behavior phenotype of the model is similar to the behavior of a typical neurodevelopment disorder animal, a brand new perspective is provided for exploring an etiology mechanism, model mice can be prepared on a large scale by performing directional mating on the gene modified mice, and the development of the model is promoted. And consistency of different experiment batches and reliable reproduction of experiment data are ensured.

A protein nanospheres and method to treat dysfunction from chemotherapy and immunosuppressive therapy, and a manufacture of fibrinogen-coated albumin spheres (FAS) and High-Fibrinogen Spheres (HFS) which have higher concentrations of fibrinogen molecules per sphere than FAS, and their use for medical treatments. Both kinds of nanoparticles are effective in the mitigation of the toxic effects of certain chemotherapeutic and radiological agents that are typically used in the treatment of cancer, or the treatment of autoimmune diseases, or for patients with both diseases. FAS and HFS can exert their beneficial effects via a variety of mechanisms which match the need of the body for specific cell types, including any of the subgroups of T cells and antibody producing cells, the relative concentration of each kind is vital to the balance between tumor surveillance and autoimmune disease suppression.

The present invention relates to polyplexes comprising linear conjugates of LPEI and PEG. The LPEI and PEG fragments of the linear conjugates are preferably linked by a [3+2]cycloaddition between an azide and an alkene or an alkyne to produce a 1, 2, 3 triazole or a 4,5-dihydro-1H-[1,2,3]triazole. The linear conjugates are further conjugated to a targeting fragment capable of binding to prostate specific membrane antigen (PSMA) to enable selective interaction with a particular cell type. The conjugates can form polyplexes with therapeutic agents such as nucleic acids to deliver the therapeutic agents to cells.

The present disclosure provides methods to genetically modify cells by insertion of an artificial exon (ArtEx) for delivery of therapeutic proteins in specific cell types and more particularly engineered cells for expression of a transgene into the brain of a patient.

Various embodiments are directed to the analysis of fragmentation patterns of cell-free DNA (cfDNA) circulating in cerebrospinal fluid (CSF) and the potential applications. CSF is an important liquid biopsy sample used to study the central nervous system and related disorders, such as infection and malignancies. The characterization of fragmentation patterns of cfDNA in CSF includes the size profile, end motif, cleavage profiles, and the determination of epigenetic features, including methylation. Various applications can use one or more properties of fragmentation pattern, for example, in the determination of the proportional contribution of a particular cell types in the CSF cfDNA pool. Another purpose is the diagnosis of pathology in the central nervous system, by the detection of clinically relevant DNA (e.g., tumor fraction, pathogen). DNA fragments in CSF can be analyzed in various ways, including using short-read sequencing, and / or long-read sequencer technologies.

The present disclosure is directed to scaffold materials patterned with cell adhesion targets that permit the attachment and growth of predetermined cell types onto the scaffold. By selecting specific ligands that are patterned on the scaffold, particular cell types, including those engineered for this specific purpose, will attach and proliferate to create designed tissues and organs.

The present invention relates to a polyplex comprising a linear conjugate of LPEI and PEG. The LPEI and PEG fragments of the linear conjugate are preferably linked by a [3+2] cycloaddition between azide and alkene or alkyne to generate 1,2,3 triazole or 4,5-dihydro-1H-[1,2,3]triazole. The linear conjugate is further conjugated to a targeting fragment that can bind to prostate-specific membrane antigen (PSMA) to enable selective interaction with specific cell types. The conjugate can form a polyplex with a therapeutic agent, such as a nucleic acid, to deliver the therapeutic agent to cells.

The present invention provides a targeting polyplex composed of (i) a nucleic acid, particularly a nucleic acid encoding a pharmaceutically active peptide or protein, and (ii) a targeting conjugate comprising an LPEI and a PEG fragment connected by distinct linkages formed by a defined chemoselective reaction. Thus, the LPEI fragment is linked to a single PEG fragment in a linear, end-to-end manner. The linear conjugate is further conjugated to a targeting fragment to enable selective interaction with specific cell types. The polyplex selectively delivers the nucleic acid to target cells, resulting in high expression and efficient protein translation and secretion of the encoded pharmaceutically active protein.

The application relates to a retinal cell type-based visual function related parameter acquisition system and belongs to the technical field of medical auxiliary systems. The application aims to accurately acquire a retinal cell type-based visual function related parameter acquisition system of L cone, M cone, S cone, ipRGCs and other different retinal cell type function related physiological parameters. The system of the application comprises a stimulation presentation module, a brightness calibration module, a biological signal acquisition module, a multi-dimensional parameter extraction and cell type specific parameter decoupling module, a parameter analysis and standardized output module, a user interaction module and a data storage module. The application can provide a portable, objective, non-invasive and cell type specific retinal function related parameter acquisition technology, provide comprehensive and reliable technical support for visual function evaluation, is suitable for large-scale parameter acquisition and home monitoring, and solves many limitations of the prior art.

Described herein are compositions and methods for potent and long-lasting gene editing in a cell. The gene editing may result in an increase in function of a gene product. The cell can be of a particular cell type, such as a basal cell, a ciliated cell, or a secretory cell. In some cases, the cell can be a lung cell of a particular cell type. The presently disclosed compositions comprise a lipid composition, which can comprise an ionizable cationic lipid, a sterol, a phospholipid, and a selective organ targeting lipid. Also described herein are methods for treating a disease or disorder, such as a lung disease or disorder, involving the presently disclosed compositions.

Materials and methods for labeling and isolating particular cell types from mixed cell populations are provided herein. Also provided herein are methods for generating data representing a synthetic genetic sequence configured for labeling at least one cell type by causing expression of a marker in the at least one cell type.

The present invention provides methods of using the RNA analysis to identify a cell type from which a midbody remnant (MBR) originated. The methods include isolating MBR, analyzing the RNA in the MBR to determine RNA transcript levels, and using the RNA transcript levels to identify a cell type from which the MBR originated. The method may be used to detect proliferative diseases including cancer by comparing RNA transcript levels to control levels.

The present invention relates to polyplexes comprising linear conjugates of LPEI and PEG. The LPEI and PEG fragments of the linear conjugates are preferably linked via a [3+2] cycloaddition between an azide and an alkene or alkyne to produce a 1,2,3 triazole or a 4,5-dihydro-1H-[1,2,3]triazole. The linear conjugates are preferably further conjugated to a targeting fragment to enable selective interaction with specific cell types. The conjugates can form polyplexes with therapeutic agents, such as nucleic acids, to deliver the therapeutic agents to cells.

The present invention relates to polyplexes comprising linear conjugates of LPEI and PEG. The LPEI and PEG fragments of the linear conjugates are preferably linked by a [3+2] cycloaddition between an azide and an alkene or an alkyne to produce a 1, 2, 3 triazole or a 4,5-dihydro-1H-[1,2,3]triazole. The linear conjugates are preferably further conjugated to a targeting fragment to enable selective interaction with a particular cell type. The conjugates can form polyplexes with therapeutic agents such as nucleic acids to deliver the therapeutic agents to cells.

To provide radiohalogen prosthetic moieties and radiolabeled biomolecules.SOLUTION: The present application relates to radiohalogen prosthetic moieties and precursors thereof and to radiolabeled biomolecules comprising such radiohalogen prosthetic moieties. The biomolecules have an affinity for particular types of cells and may specifically bind a certain cell, such as a cancer cell. Relevant biomolecules include antibodies, monoclonal antibodies, antibody fragments, peptides, other proteins, nanoparticles, aptamers, and pharmacological moieties used to target prostate-specific membrane antigen (PSMA).SELECTED DRAWING: None

The invention provides a pathological section data feature description method based on self-supervised learning. The method comprises the following steps: dividing pathological sections into a plurality of groups of pathological sections in a manner that two pathological sections form one group; splicing the weighted first pathological feature and the weighted second pathological feature to obtain a cross attention feature map, and obtaining a probability value that the group of pathological sections are the same patient through a full connection layer; processing the cross attention feature map through a segmentation network to obtain a binary mask of a specific cell type; and according to the binary mask, the patient label and the probability value, calculating a dynamic loss function and a dynamic learning rate optimization model feature. By adopting the method, the whole self-supervised learning process does not need to depend on an external task label, and feature learning is carried out only based on the pathological section. The marking cost is effectively reduced, and an efficient and low-cost solution is provided for feature description of the pathological image.

The present disclosure relates to a molecular delivery system that facilitates the internalization of LNPs into a specific target of choice, such as a specific cell type, ex vivo and / or in vivo. The present disclosure also relates to methods, molecules, and compositions for enhancing the targeted delivery of compounds within a living system. In particular, embodiments provided herein relate to methods, molecules, and compositions for the targeted delivery of lipid nanoparticles containing therapeutic molecules into a cell or system of choice, such as a T cell. The present disclosure also relates to methods of administering the enhanced targeting system to a patient or system, compositions for use in such methods, and further methods of use of the targeting system as part of T cell-based immunotherapy.

The present invention provides targeting polyplexes comprised of (i) nucleic acids, in particular nucleic acids encoding pharmaceutically active peptides or proteins, and (ii) targeting conjugates comprising LPEI and PEG fragments that are connected by discrete linkages formed through defined, chemoselective reactions. Thus, the LPEI fragment is bonded in a linear end- to-end fashion to a single PEG fragment. The linear conjugates are further conjugated to a targeting fragment to enable selective interaction with a particular cell type. The polyplexes selectively deliver the nucleic acids to the targeted cells resulting in high expression and efficient protein translation as well as secretion of the encoded pharmaceutically active proteins.

To provide a cell-targeting system that delivers a therapeutic nucleic acid to a specific cell type and to a specific location within the cell.SOLUTION: A composition comprising a nucleic acid sequence conjugated to one or more molecular guidance system (MGS) peptides.SELECTED DRAWING: Figure 1

The present invention relates to Shiga toxin effector polypeptides with reduced antigenic and / or immunogenic potential. Immunogenicity can be a limitation for the repeated administration to mammals of proteins and polypeptides derived from Shiga toxins. The Shiga toxin effector polypeptides of the present invention have uses as components of therapeutics, diagnostics, and immunization materials. The cytotoxic proteins of the present invention have uses for selective killing of specific cell types and as therapeutics for the treatment of a variety of diseases, including cancers, immune disorders, and microbial infections. The proteins of the present invention also have uses for detecting specific cell types, collecting diagnostic information, and monitoring the treatment of a variety of diseases, such as, e.g., cancers, immune disorders, and microbial infections.

To fully understand the biology of the brain, different cell types need to be distinguished and defined and, to further study them, artificial expression constructs that can label and perturb them need to be identified, in mouse, recombinase driver lines have been used to great effect to label cell populations that share marker gene expression. However, the creation, maintenance, and use of such lines that label cell types with high specificity can be costly, frequently requiring triple transgenic crosses, which yield a low frequency of experimental animals. Artificial expression constructs for modulating gene expression in targeted central nervous system cell types are described. The artificial expression constructs can be used to express synthetic genes or modify gene expression in non-neuronal cells including oligodendrocytes, microglia, astrocytes, or endothelial cells.

This invention belongs to the field of pharmaceutical technology and discloses a pharmaceutical composition of a USP7 inhibitor and fenofibrate and its antitumor application. The pharmaceutical composition uses a USP7 inhibitor and fenofibrate as the core active ingredients, with a molar ratio of 0.015625 to 0.625. The USP7 inhibitor is selected from at least one of LX04-104 and LML-17-133. This invention is the first to discover that fenofibrate can significantly enhance the antitumor effect of the USP7 inhibitor, and the combination of the two has a synergistic effect. This synergistic effect is cell type specific, particularly significant for nasopharyngeal carcinoma and pancreatic cancer, and the synergistic effect of fenofibrate is independent of the PPARα pathway. This composition can be prepared as an oral or injectable formulation for the treatment of nasopharyngeal carcinoma and pancreatic cancer, and has good prospects for clinical translation.

The present invention provides a method for processing biological sample information, comprising the following steps: obtaining transcriptome information of a biological sample to be tested and a plurality of known reference biological samples; based on the transcriptome information, performing a global analysis based on whole genes, a local analysis based on differential genes, a feature analysis based on gene differential expression analysis, and a non-negative least squares regression analysis on the biological sample to be tested and the plurality of reference biological samples; and determining the overall characteristics of the biological sample to be tested based on the results of the global analysis, the local analysis, the feature analysis, and the non-negative least squares regression analysis. Based on high-throughput sequencing data, the present invention studies and establishes a set of methods that can be used to systematically evaluate the similarities between biological samples such as cells and tissues, and based on this method establishes a research strategy for determining the stage and state of a cell lineage, providing reference value for the specific positioning of specific cell types and subpopulations in cells of different lineages.

Lipid nanoparticle formulations with cell type specific transfection activity and capable of producing Th1 and / or Th2 response in vivo and their use for plasmid DNA or mRNA delivery is disclosed.

A method for predicting the metabolic state of a cell culture of cells of a specific cell type includes providing a metabolic model of a cell of the specific cell type, and performing at each of a plurality of points in time during cultivation of the cell culture, receiving measured concentrations of a plurality of extracellular metabolites and a measured cell density in the culture medium; inputting the received measurements as input parameter values to a trained machine learning program logic-MLP; predicting extracellular fluxes of the extracellular metabolites at a future point in time by the MLP; performing metabolic flux analysis to calculate the intracellular fluxes at the future point in time based on the predicted extracellular fluxes and the stoichiometric equations of the metabolic model.

Various embodiments are directed to the analysis of fragmentation patterns of cell-free DNA (cfDNA) circulating in cerebrospinal fluid (CSF) and the potential applications. CSF is an important liquid biopsy sample used to study the central nervous system and related disorders, such as infection and malignancies. The characterization of fragmentation patterns of cfDNA in CSF includes the size profile, end motif, cleavage profiles, and the determination of epigenetic features, including methylation. Various applications can use one or more properties of fragmentation pattern, for example, in the determination of the proportional contribution of a particular cell types in the CSF cfDNA pool. Another purpose is the diagnosis of pathology in the central nervous system, by the detection of clinically relevant DNA (e.g., tumor fraction, pathogen). DNA fragments in CSF can be analyzed in various ways, including using short-read sequencing, and / or long-read sequencer technologies.

Described herein are compositions and methods for potent and long-lasting gene editing in a cell. The gene editing may result in an increase in function of a gene product. The cell can be of a particular cell type, such as a basal cell, a ciliated cell, or a secretory cell. In some cases, the cell can be a lung cell of a particular cell type. The presently disclosed compositions comprise a lipid composition, which can comprise an ionizable cationic lipid, a sterol, a phospholipid, and a selective organ targeting lipid. Also described herein are methods for treating a disease or disorder, such as a lung disease or disorder, involving the presently disclosed compositions.

The invention relates to a method for determining physically interacting cells in a cell population. The method comprises (a.) providing a flow-cytometry data set of said cell population, said data set comprising data on scattering properties and expression of cell-type specific surface markers for the cells in said cell population, wherein said scattering properties comprise at least one measurement of forward and / or side scatter, (b.) applying an automated clustering algorithm to said flow-cytometry data set, thereby assigning the cells to a cluster according to a degree of similarity of the scattering properties and the expression of cell-type specific surface markers, (c.) wherein the expression of one or more cell-type specific surface marker(s) and / or combinations of cell-type specific surface markers in a cluster is indicative of a specific cell type(s) in the cluster, and wherein the scattering properties of a cluster are indicative of the presence of a single cell or two or more physically interacting cells in the cluster. The invention further relates to software configured for a method to determine physically interacting cells in a cell population from a flow-cytometry data set and a computer-readable storage device, comprising the software.