2938results about "Cell receptors/surface-antigens/surface-determinants" patented technology

Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload inhibits expression or activity of a DMPK allele comprising a disease-associated-repeat. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.

The invention provides a method of treating an adult subject having a hematological cancer, comprising administering to the subject selected dosage regimens comprising a plurality of immune effector cells expressing a CAR molecule.

The disclosure provides a method of treating a human subject afflicted with lung cancer (e.g., non-small cell lung cancer (NSCLC)) with a programmed death-1 (PD-1) pathway inhibitor (e.g., an anti-PD-1 antibody) and a concurrent chemoradiotherapy (CCRT, e.g., a platinum doublet chemotherapy (PDCT) and a radiation therapy) followed by a combination of a PD-1 pathway inhibitor (e.g., an anti-PD-1 antibody) and a lymphocyte activation gene-3 (LAG-3) antagonist (e.g., an anti-LAG-3 antibody). In some aspects, the method comprises a recovery period that begins upon completion of the treatment with the PD-1 pathway inhibitor and the CCRT and ends at the start of the treatment with the combination of the PD-1 pathway inhibitor and the LAG-3 antagonist.

The present invention discloses a nucleic acid aptamer binding to CD117 protein and its application, wherein the nucleic acid aptamer has a nucleotide sequence that has at least 60% homology with any one of SEQ ID NO.1 to SEQ ID NO.2 and binds to CD117 protein. The nucleic acid aptamer of the present invention has a small molecular weight, stable structure and chemical properties, is easy to store and label, is easy to modify, and can be artificially synthesized in a short period of time. The nucleic acid aptamer obtained by the present invention can be used for CD117 protein detection, purification or imaging, and can also be used to prepare drugs for diagnosis or treatment of abnormal expression of CD117 protein, such as purification to obtain high-sensitivity and high-specificity detection of CD117 protein, and has broad application prospects.

The embodiment of the invention is to conformationally constrain neutralizing B cell epitopes of CoV-2, CoV-n B cell epitopes of the open reading frame proteins, including the membrane S protein, constrained by protein scaffolds, including camelid VHH nanobody in order to elicit neutralizing and blocking antibodies against viral infection. B cell epitopes are molecularly evolved by ribosome display to preempt emerging viral B cell epitopes. Moreover, CTL epitopes are designed from the open reading frame protein for eliciting cell mediated viral protection. Furthermore, mRNA vaccines are prepared for eliciting protective immunity and prevent cytokine storms.

Disclosed herein are compositions of retroviruses and methods of using the same for gene delivery, wherein the retroviruses comprise a viral envelope protein comprising at least one mutation that diminishes its native function, a non-viral membrane-bound protein comprising a membrane-bound domain and an extracellular targeting domain.

The present application relates to a TIGIT-derived polypeptide or a mutant thereof, and a fusion protein, an engineered receptor or an engineered cell comprising the polypeptide or the mutant, wherein the binding capacity of the TIGIT-derived polypeptide or the mutant thereof with CD112 is reduced.

Disclosed are compositions containing exosomes loaded with arginase or arginase chimeras for treating hyperammonemia due to arginase deficiency, and methods for treating hyperammonemia by administering compositions containing exosomes loaded with arginase or arginase chimeras.

The present disclosure relates to chimeric Tim4 receptors, host cells modified to include chimeric Tim4 receptor molecules, and methods of making and using such receptor molecules and modified cells.

The present invention relates to pluripotent stem cell comprising an expression construct for expression of a CEBPα protein and an expression construct for expression of an ADIG protein. The invention further provides for methods of producing adipocytes comprising the pluripotent stem cells and for foodstuff comprising the adipocytes or pluripotent stem cells.

Provided are methods for delivering lipid nanoparticles (LNPs) to a lung cell of a subject suffering from or at risk for primary ciliary dyskinesia (PCD), wherein the method comprises nebulizing a liquid pharmaceutical composition to generate an aerosolized pharmaceutical composition, and administering the aerosolized pharmaceutical composition to the subject.

Provided are a method and the like for efficiently screening for a candidate substance for a starting material that suppresses hexanal odor. The screening method according to the present invention comprises the steps of: adding a test substance and hexanal to a specific olfactory receptor polypeptide; a step for measuring the response of the olfactory receptor polypeptide to hexanal; and identifying, on the basis of the measured response, a test substance that inhibits the response of the olfactory receptor polypeptide as a candidate substance for a raw material that inhibits hexanal odor.

Aspects of the invention are drawn to compositions and methods for treating and preventing autoimmune disease.

The present disclosure, in some aspects, relates to antisense oligonucleotides (ASO) for use in the prevention or treatment of a disease or a condition associated with low- density lipoprotein (LDL) in a subject. In some embodiments, the ASO effects site-directed adenosine-to-inosine (A-to-l) editing of a target adenosine in a target RNA sequence derived from a sequence of an endogenous low-density lipoprotein receptor (LDLR) gene such that: a) the modified LDLR protein has: (i) reduced binding to the inducible degrader of the LDLR protein (IDOL); (ii) increased stability; (iii) improved resistance to IDOL-mediated degradation; (iv) increased LDLR protein expression; and / or (v) increased activity or function to take up LDL; and / or b) editing of the 3'-untranslated region (UTR) of the target RNA leads to an increase in LDLR protein expression and / or stability.

The invention relates to a YaxAB nanopore, a system comprising the nanopore and application of the YaxAB nanopore. The YaxAB nanopore of the present invention has a funnel-shaped three-dimensional structure so that the change in current blocking according to the movement of an analyte captured inside the nanopore can be maximized, thereby enabling very sensitive measurement even of the structure and minute changes in physical and chemical properties (including dynamic changes and the like) of the analyte. And can cause very strong electroosmosis with high cation selectivity, such that various types of analytes can be captured and analyzed without being limited to the type of total charge. In addition, with respect to the nanopore of the present invention, the amino acid sequence of the monomer constituting the nanopore can be adjusted to improve protein purification yield and control an oligomerization mechanism, and thus a YaxAB nanopore having a pore size that cannot be formed by a wild-type monomer can be prepared. Furthermore, with respect to the nanopores of the present invention, YaxAB nanopores of various pore diameters can be secured using one-time purification, and nanopores of desired pore diameters can be selectively secured. Thus, limitations of conventional biological nanopores having fixed pore diameters can be overcome, so that for the YaxAB nanopore of the present invention, the size of the nanopore can be adjusted according to the size of an analyte. Further, the YaxAB nanopores of the present invention have a very wide analyte analysis range of dynamic detection range from small analytes to large analytes, which is caused by a funnel-shaped three-dimensional structure having a wide inlet and a narrow outlet. In addition, the YaxAB nanopore of the present invention has the largest nanopore structure among the hitherto biological nanopores, and a large-size analyte, which cannot be detected by conventional biological nanopores, can be detected and analyzed in a single molecule on the basis of a funnel structure having a large inlet. Furthermore, the present invention has established an optimized composition of a membrane in which YaxAB nanopores can be stably present, and by using a sensor implemented on the basis thereof, it is possible to qualitatively and quantitatively analyze various types of analytes having relatively various sizes and charges at a single molecule level with very high resolution and sensitivity.

The present disclosure provides materials and methods for delivery of a transgene to target cells. The regulatory elements, transgenes, and expression vectors are useful in, e.g., expressing a transgene in CNS cells that results in an improvement in at least one symptom related to a neurological disease or disorder, including epilepsy disorders, such as refractory epilepsy.

Compositions of retroviruses and methods of using the same for gene delivery, wherein the retroviruses comprise a viral envelope protein comprising at least one mutation that diminishes its native function, a non-viral membrane-bound protein comprising a membrane-bound domain and an extracellular targeting domain.

Compositions comprising multiple pHLIP® peptides linked to a lipid nanoparticle encapsulating one or more nucleic acids (pHLIP®-LNP) and methods of preparing the pHLIP®-LNPs are described.

The present invention discloses a nucleic acid aptamer binding to CD5 protein, the sequence of which is shown in SEQ ID NO.1 to SEQ ID NO.6, and by improving the screening conditions, a nucleic acid aptamer with small molecular weight, stable chemical properties, easy storage and labeling, which can bind to CD5 protein with high affinity, is screened and obtained. The aptamer is measured by surface plasmon resonance (SPR) and dot hybridization experiments to have high affinity and high specificity, and can be used in detection, diagnosis, imaging and treatment, and has broad application prospects.

Genetically modified cells that are compatible with multiple subjects, e.g., universal donor cells, and methods of generating the genetic modified cells are provided herein. The universal donor cells comprise at least one genetic modification within or near at least one gene that encodes one or more MHC-I or MHC-II human leukocyte antigens or component or transcriptional regulator of the MHC-I or MHC-II complex, at least one genetic modification that increases the expression of at least one polynucleotide that encodes a tolerogenic factor, and optionally at least one genetic modification that increases or decreases the expression of at least one gene that encodes a survival factor.

The invention relates to a method for carrying out gene editing on a TRAC gene locus. Specifically, the invention provides sgRNA targeting and guiding nuclease to efficiently cut a TRAC gene, a method for modifying a CAR-T cell by using the sgRNA, the CAR-T cell obtained by the method, and a related gene editing system, reagent and kit. According to the CAR-T cell, the immunogenicity is reduced, the risk of graft versus host disease and immunological rejection can be effectively reduced, and meanwhile the killing capacity of tumor cells of the CAR-T cell is not affected.

Genetically modified cells that are compatible with multiple subjects, e.g., universal donor cells, and methods of generating the genetically modified cells are provided herein. The universal donor cells comprise at least one genetic modification within or near at least one gene that encodes one or more MHC-I or MHC-II human leukocyte antigens or component or transcriptional regulator of the MHC-I or MHC-II complex, at least one genetic modification that increases the expression of at least one polynucleotide that encodes a tolerogenic factor, and optionally at least one genetic modification that increases or decreases the expression of at least one gene that encodes a survival factor.

The present disclosure provides recombinantly manufactured fusion proteins comprising a Programmed Death-Ligand 1 (PD-L1) protein fragment or an analog thereof linked to a canine Fc fragment. Embodiments include the administration of the fusion proteins to patients as a treatment for cancers, tumors or other diseases associated with expression of the PD-L1 protein in dogs. Exemplary Fc fusion proteins and pharmaceutical formulations of exemplary Fc fusion proteins are provided, in addition to methods of use and preparation.

Disclosed herein are isolated polypeptide and polypeptide complex compositions that comprise a first antigen recognizing molecule that binds to CD28 and a second antigen recognizing molecule that binds to prostate-specific membrane antigen (PSMA).

To provide induced regulatory T cells with high function and stable immunosuppressive function.SOLUTION: The present invention provides an induced human regulatory T cell having at least one characteristic selected from the group consisting of FoxP3-positive, CTLA4-positive, NT5E-positive, ITGAE (CD103)-positive, AREG-positive, CD172 g-positive, and CD26-positive, which is induced by obtaining stable inducible T cells induced from human peripheral T cells by stimulation with an anti-CD3 antibody when regulatory T cells are induced from human peripheral T cells, subsequently subjecting the cells to resting culture, stimulating the cells with anti-CD3 antibody again to culture them, and further subjecting the cells to resting culture.SELECTED DRAWING: None

Genetically engineered immune cells, which express at least two metabolism modulating polypeptides and optionally a chimeric receptor polypeptide (e.g., an antibody-coupled T cell receptor (ACTR) polypeptide or a chimeric antigen receptor (CAR) polypeptide) capable of binding to a target antigen of interest. Also disclosed herein are uses of the engineered immune cells for inhibiting cells expressing a target antigen in a subject in need thereof.

The present invention provides, in certain aspects, a natural killer (NK) cell that expresses all or a functional portion of interleukin-15 (IL-15), and methods for producing such cells. The invention further provides methods of using a natural killer (NK) cell that expresses all or a functional portion of interleukin-15 (IL-15) to treat cancer in a subject or to enhance expansion and / or survival of NK cells.

The present invention provides methods and compositions for treating cancer and / or autoimmune diseases by modulating the expression and / or activity of IGSF8 and its binding ligand. Pharmaceutical compositions include, but are not limited to, antibodies that specifically bind to human IGSF8 and have the activity of inhibiting IGSF8-mediated immunosuppression in a subject in need thereof. Pharmaceutical compositions may include, but are not limited to, antibodies that specifically bind to human IGSF8 and have the activity of inhibiting IGSF8-mediated immunosuppression in a subject in need thereof.

The present invention relates to antibodies, and fragments thereof, that bind and inhibit CD73. The invention also relates to cells producing such compounds; methods of making such compounds, and antibodies, fragments, variants, and derivatives thereof; pharmaceutical compositions comprising the same; methods of using the compounds to diagnose, treat or prevent diseases, e.g., cancer.

The invention relates to the field of therapeutics and, more in particular, it concerns the use of a low-density lipoprotein receptor-related protein 5 (LRP5) inhibitor for the treatment and / or prevention of thrombosis in a patient as well as genetically modified megakaryocyte or progenitor thereof in which LRP5 gene expression has been silenced and the uses thereof for the treatment and / or prevention of thrombosis in a patient.