46 results about "Cell type specific" patented technology

Described herein are compositions, kits, and methods for potent delivery to a cell of a subject. 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. Also described herein are pharmaceutical compositions comprising a therapeutic or prophylactic agent assembled with a lipid composition. The lipid composition can comprise an ionizable cationic lipid, a phospholipid, and a selective organ targeting lipid. Further described herein are high-potency dosage forms of a therapeutic or prophylactic agent formulated with a lipid composition.

The disclosure relates to growing cells, directing cells to grow into specified cell types, genetically and physically manipulating cells, and addressing one or more individual cells within a mixed cell population. Aspects of the disclosure relate to vectors useful to induce developmental changes in cells, in which those vectors have a temporal component. Vectors of the disclosure encode a controllable, temporal series of events. Once the vectors are delivered into target cells, a series of discrete and different genetic events may be induced. The disclosed methods generally provide for the temporal encoding of multiplex genetic effectors in vector format for cell state transitions.

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.

Provided are isolated, cloned enhancer element sequences that target certain neuronal cell types, subtypes, and / or populations in the brain and CNS. The isolated and cloned enhancer elements can target a gene of interest, such as a therapeutic gene or an effector gene, to be expressed in the certain cell types. Virus vectors containing the isolated, cloned enhancer element sequences, particularly, recombinant adeno-associated virus (rAAV) vectors, and a gene or polynucleotide to be expressed in a given neuronal cell type are described. The rAAV vectors, compositions and methods are useful for treating subjects afflicted with neuropsychiatric and neuropathological diseases, disorders and conditions and symptoms thereof.

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 polymer complexes comprising linear conjugates of LPEI and PEG. The LPEI fragment and the PEG fragment of the linear conjugate are preferably connected through [3 + 2] cycloaddition reaction between azide and olefin or alkyne to generate 1, 2, 3 triazole or 4, 5-dihydro-1H-[1, 2, 3] triazole. The linear conjugates are preferably further conjugated to targeting fragments to achieve selective interaction with specific cell types. The conjugate may form a polymer complex with a therapeutic agent (e.g., a nucleic acid) to deliver the therapeutic agent to a cell.

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 disclosure relates to growing cells, directing cells to grow into specified cell types, genetically and physically manipulating cells, and addressing one or more individual cells within a mixed cell population. Aspects of the disclosure relate to vectors useful to induce developmental changes in cells, in which those vectors have a temporal component. Vectors of the disclosure encode a controllable, temporal series of events. Once the vectors are delivered into target cells, a series of discrete and different genetic events may be induced. The disclosed methods generally provide for the temporal encoding of multiplex genetic effectors in vector format for cell state transitions.

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 present invention provides targeted polymer complexes comprising: (i) a nucleic acid, in particular a nucleic acid encoding a peptide or protein having pharmaceutical activity; and (ii) a targeting conjugate comprising LPEI and PEG fragments linked by discrete bonds formed by a defined chemically selective reaction. Thus, the LPEI fragment is bonded to a single PEG fragment in a linear end-to-end manner. The linear conjugate is further conjugated to a targeting fragment to achieve selective interaction with a particular cell type. The polymer complexes selectively deliver nucleic acids to target cells, thereby achieving high expression and efficient protein translation and secretion of the encoded pharmaceutically active proteins.

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.

The inventors developed a new system to modify tropism of syncytins, envelope proteins, which can be used to functionalize particles such as virus particle or more particularly viral-like particles (VLPs), and used for gene transfer or other applications. In particular, they created a fusion protein containing the Syncytin-1 (SYN) signal sequence (SS), a targeting moiety (either natural or engineered), the SYN protein and a flexible linker between SYN and the targeting moiety to enhance transduction of the cell type expressing the receptor or the antigen targeted by the targeting moiety, for instance hematopoietic stem progenitor cells (HSPCs). The inventors demonstrated that the fusion strategy allows modification of syncytin tropism towards different receptors in order to target f the desired cell type. The system is adaptable to other desired antigens to retarget the fusion protein to a specific cell type.

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 predicting the metabolic condition of a cell culture of cells of a specific cell type. The method comprises providing (102) a metabolic model (402) of a cell of the specific cell type and carrying out the following steps at each of a plurality of points in time during the cultivation of the cell culture: receiving (106) measured concentrations of several extracellular metabolites and a measured cell density in the culture medium; inputting (108) the received measured values as input parameter values into a trained machine learning program logic—MLP (218); predicting (110) extracellular flows (408) of the extracellular metabolites at a future point in time by means of the MLP; carrying out (112) a metabolic flow analysis for calculating the intracellular flows at the future point in time on the basis of the predicted extracellular flows and the stoichiometric equations of the metabolic model.

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.

Provided herein are compositions and methods that allow for the identification of specific cell types transduced by barcoded adeno-associated virus (AAV). These compositions and methods are useful for identifying the tropism of AAVs having modified capsid proteins comprising targeting peptides.

Provided for herein are viral particles comprising a heterologous viral glycoprotein and a targeting moiety, wherein the targeting moiety comprises a polypeptide comprising a formula of T-S1, wherein T is a target binding domain and S1 is a stalk portion. The stalk portion may comprise a variant Fc domain. The stalk portion may comprise a flexible polypeptide domain. The targeting moiety comprising the formula T-S1 may be incorporated into a viral particle to assist with targeting such particles to a specific cell type. Also provided for herein are compositions comprising the same, and methods of using the same.

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.