15 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.

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 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 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.

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 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.

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.

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 present invention discloses methods and compositions for the identification of one or more ultra-specific phase-based methylation patterns (uPMPs) of a particular tissue or cell type, and methods and compositions for the identification of one or more ultra-specific phase-based methylation patterns (uPMPs) of a particular tissue or cell type, and methods and compositions for the identification of one or more ultra-specific phase-based methylation patterns (uPMPs) of a particular tissue or cell type. Also disclosed and described are uPMPs for certain specific tissue or cell types, as well as the use of uPMPs in diagnostics, such as incorporation thereof into a detection panel.

Described herein are polynucleotide compositions comprising 5' untranslated region (UTR) sequences that enhance translation of a payload under translational control of the 5' UTR. These 5' UTRs can enhance translation in various cell types, with some showing particular efficacy in specific cell types such as retinal pigment epithelium (RPE) cells relative to non-RPE cells. Also described herein are methods of expressing a payload in various target tissues, including but not limited to RPE, using a polynucleotide composition comprising the 5' UTR. The invention further provides libraries of 5' UTR sequences, methods for screening these libraries in multiple cell types, and recombinant polynucleotides incorporating the 5' UTRs for enhanced payload expression in various target cells, including RPE cells. These compositions and methods may be used for treating a wide range of disorders, with particular promise shown for retinal disorders through improved gene therapy approaches.