Abstract: The invention relates to an expression system comprising polynucleotides encoding proteins, wherein the expression system comprises a first polynucleotide encoding at least one protein, peptide or variant thereof, which induces a T cell response, and a second polynucleotide encoding at least one protein, peptide or variant thereof, which induces an anti-pathogenic B cell response. The invention further relates to protein mixtures encoded by the expression system and cells comprising the expression system or the protein mixture and pharmaceutical compositions comprising the expression system or the protein mixture.

Abstract: The present disclosure provides one or more amino lipids such as an amino lipids containing a sulfonic acid or sulfonic acid derivative of the formulas: wherein the variables are as defined herein. These amino lipids may be used in compositions with one or more helper lipids and a nucleic acid therapeutic agent. These compositions may be used to treat a disease or disorder such as cancer, cystic fibrosis, or other genetic diseases.

Abstract: The present disclosure provides a replication-competent, recombinant oncolytic vaccinia virus, compositions comprising the vaccinia virus, and use of the vaccinia virus or composition for inducing oncolysis in an individual having a tumor.

Abstract: The present invention relates to the use of gasdermin, in particular of gasdermin A, gasdermin B, gasdermin C, gasdermin D, DFNA5 or DFNB59 (or pejvakin), and more particularly pejvakin for modulating cellular redox homeostasis. A particularly preferred use of gasdermin, in particular of gasdermin A, gasdermin B, gasdermin C, gasdermin D, DFNA5 or DFNB59 (or pejvakin), and more particularly pejvakin in the context of the present invention is as an antioxidant. The present invention also concerns a virally-mediated gene therapy for restoring genetically-impaired auditory and vestibular functions in subjects suffering from an Usher syndrome. More precisely, this gene therapy takes advantage of an AAV2/8 vector expressing at least one USH1 gene product, preferably SANS.

Abstract: The disclosure relates to a manufactured composition, material or device comprising at least two different nonradioactive isotope atoms. Each nonradioactive isotope atom is present in an amount sufficient to increase the total amount of the nonradioactive isotope atom above the total amount found in the manufactured composition, material or device in the absence of adding the nonradioactive isotope atom to increase said total amount. The ratio(s) of the at least two nonradioactive isotopes in the manufactured composition, material or device are measurably different than the ratio(s) found in the manufactured composition, material or device in the absence of adding the nonradioactive isotope atom to increase said total amount.

Abstract: Methods and composition for modulating a target genome and stable integration of a transgene of interest into the genome of a cell are disclosed.

Abstract: Methods for treating joint diseases, disorders, and conditions, such as osteoarthritis, with a composition containing a therapeutically effective amount of tissue inhibitors of matrix metalloproteinases (TIMPs) are provided. The composition can contain TIMPs as part of amniotic fluid, or the TIMPs can be isolated from another source or recombinantly produced. The methods are particularly useful for treating osteoarthritis associated with degradation of articular cartilage by proteases. Also provided are methods of lubricating a joint, such as an osteoarthritic joint.

Abstract: Provided are a peptide having skin-whitening activity and use thereof, and a peptide consisting of an amino acid sequence of SEQ ID NO: 1, SEQ ID NO: 2, or SEQ ID NO: 3, a skin-whitening composition including the peptide, a cosmetic composition including the skin-whitening composition, and a pharmaceutical composition for preventing or treating a melanin hyperpigmentation disease, the pharmaceutical composition including the skin-whitening composition.

Abstract: An object of the invention is to provide a method for producing a mesenchymal stem cell in which differentiation into an adipocyte is suppressed with a simple step, a mesenchymal stem cell, a method for producing a differentiation-induced cell using the method for producing a mesenchymal stem cell described above, a differentiation-induced cell, and an inhibitor of differentiation into an adipocyte. According to the invention, there is provided a method for producing a mesenchymal stem cell, including: a step of culturing a mesenchymal stem cell in a liquid medium in which a recombinant gelatin having an amino acid sequence derived from a partial amino acid sequence of collagen has been dissolved, wherein differentiation into an adipocyte is suppressed.

Abstract: Disclosed herein are methods and compositions comprising an adeno-associated virus 2.5 (AAV2.5) capsid protein, comprising one or more amino acids substitutions, (e.g., which does not contain a substitution at the position corresponding to amino acid 267 of AAV2.5, or does not contain a serine at the position corresponding to amino acid 267 of AAV2.5) wherein the substitutions introduce a new glycan binding site into the AAV capsid protein.

Abstract: The present disclosure relates a polypeptide having UGT activity, which polypeptide comprises an amino acid sequence which, when aligned with a polypeptide having UGT activity comprising the sequence set out in SEQ ID NO: 2, comprises at least one substitution of an amino acid corresponding to any of amino acids at positions 35, 189, 280, 284, 285, 334 or 373, said positions being defined with reference to SEQ ID NO: 2 and wherein the polypeptide has one or more modified properties as compared with a reference polypeptide having UGT activity. A polypeptide according to the disclosure may be used in a recombinant cell for the production of steviol or a steviol glycoside.

Abstract: The present invention is directed to promoter sequences and promoter control elements, polynucleotide constructs comprising the promoters and control elements, and methods of identifying the promoters, control elements, or fragments thereof. The invention further relates to the use of the present promoters or promoter control elements to modulate transcript levels.

Abstract: Methods, compositions, and systems are provided that allow for reliable sequencing of the initial sequence region of a sequence of interest. The methods of the invention allow for more reliable barcoding of subpopulations of nucleic acids to be sequenced.

Abstract: The invention concerns a multicistronic nucleic acid, in particular an isolated multicistronic nucleic acid, comprising: A) a sequence comprising successively: A1) a sequence encoding the light chain variable domain of an antibody of interest, fused in the frame with A2) a sequence encoding the constant region of the light chain of an immunoglobulin Ig; and B) a sequence comprising successively: B1) a sequence encoding the heavy chain variable domain of said antibody of interest, fused in the frame with B2) a sequence encoding the constant regions of the heavy chain of an immunoglobulin Ig? in secretory form; B3) an intronic sequence of the gene of the heavy chain of said immunoglobulin Ig?, said intronic sequence comprising an internal 5? splice site enabling the splicing of said intronic sequence B3) and a secretory-specific poly(A) (p AS) signal from the 3? terminal exon of said gene; B4) a sequence, in frame with sequence B1), encoding the transmembrane and cytoplasmic domains M1 and M2 of the immunoglobu

Abstract: Provided herein are compounds, compositions and methods for balancing a T-helper cell profile and in particular Th1, Th2, Th17 and Treg cell profiles, and related methods and compositions for treating or preventing an inflammatory condition associated with an imbalance of a T-helper cell profile.

Abstract: A monocyte, monocyte derived cell or macrophage infected with an oncolytic herpes simplex virus is disclosed together with uses of such infected cells in the treatment of diseases such as cancer.

Abstract: This disclosure provides ASCT2-binding molecules, e.g., anti-ASCT2 antibodies, and antigen-binding fragments thereof. In certain aspects, the ASCT2-binding molecules are conjugated to cytotoxic drugs, e.g., ASCT2 antibody-drug conjugates (ADCs). In certain aspects, the anti-ASCT2 antibodies and fragments thereof can be hybridoma-derived murine monoclonal antibodies, and humanized versions thereof. In certain aspects, the ASCT2-binding molecules bind specifically to cells expressing ASCT2, and in some instances, are internalized into the cells. In addition, this disclosure provides compositions and methods for diagnosing and treating diseases or disorders characterized by ASCT2 overexpression, e.g., certain types of cancer. In a particular embodiment, the disclosure provides methods for treating cancer using ASCT2 ADCs.

Abstract: The present invention aims to provide a method for simply and highly accurately detecting castration-resistant prostate cancer (CRPC), and a reagent that can be used for this method. By measuring the level of GDF15 propeptide present in a sample as a novel detection marker for CRPC, acquisition of castration resistance in a prostate cancer patient during or after endocrine therapy is detected. An antibody that specifically recognizes GDF15 propeptide is included in the CRPC detection reagent.

Abstract: The present disclosure provides improved genome editing compositions and methods for editing a TCR? gene.

Abstract: The present invention provides peptides and peptide-conjugates that bind to ?v?6 integrin. The peptide-conjugates can be used for a variety of imaging and therapeutic applications. Methods of use and peptide optimization are also provided herein.

Abstract: The present invention relates to compositions and methods for identifying, assessing, preventing, and treating melanoma. A variety of PD-L1 isoform biomarkers are provided, wherein alterations in the copy number of one or more of the biomarkers and/or alterations in the amount, structure, and/or activity of one or more of the biomarkers is associated with melanoma status.

Abstract: Novel insecticidal proteins that are toxic to lepidopteran pests are disclosed. The DNA encoding the insecticidal proteins can be used to transform prokaryotic and eukaryotic organisms to express the insecticidal proteins. The recombinant organisms or compositions containing the recombinant organisms or the insecticidal proteins alone or in combination with an appropriate agricultural carrier can be used to control lepidopteran pests in various environments.

Abstract: The present invention relates to systems and methods for tissue processing and imaging including a counterintuitive inverse relationship between protein dye concentration and tissue sample protein content. Varying dye concentration in such a manner affords higher quality fluorescent imaging at depth in tissue when using optical sectioning microscopy or second harmonic generation (SHG) analysis. Methods of the invention thereby provide more usable histopathology images while reducing waste and reagent costs and are of particular utility in combination assays that include simultaneous protein and nuclear staining and SHG analysis.

Abstract: The invention relates to methods for enzymatic, genotyping of polymorphisms on solid supports. In some aspects, the methods include ligation of allele or base specific 5? interrogation probes to an array probe. The array probe is labeled by ligation of the interrogation probe. Ligation is dependent on the identity of the base immediately adjacent to the end of the array probe. In other aspects array bound probes are labeled by template dependent extension.

Abstract: A transfection reagent composition comprising: 30-60 MOL. % of a cationic lipid, or a pharmaceutical acceptable salt thereof; 10-60 MOL % of a structural lipid; 10-20 MOL % of a triglyceride; and 0.1 to about 10 MOL. % of a stabilizing agent, is provided. The transfection agent is effective in transfecting cells, particularly neurons, with siRNA, mRNA and plasmid nucleic acid, Sand maintaining viability of the cells as well as activity of the delivered nucleic acid.

Abstract: The present invention discloses a mutant of cyclodextrin glycosyltransferase and belongs to the fields of gene engineering and enzyme engineering. According to the present invention, a mutant having higher disproportionation activity of cyclodextrin glycosyltransferase is obtained by mutating the cyclodextrin glycosyltransferase. The disproportionation activity of enzymes of mutants V6D, S90G, T168A, T171A, T383A, G608A, and V6D/S90G/T168A/T171A/T383A/G608A, is respectively 1.89 times, 1.21 times, 1.21 times, 1.22 times, 1.32 times, 2.03 times, and 3.16 times that of the wild type enzyme in shake flask fermentations.

Abstract: Devices and methods for performing pre-analysis sample processing of biological and chemical samples using robotic liquid handlers are disclosed. Methods for solid phase extraction, protein precipitation and filtration of biological and chemical samples using automation and the devices in a rapid and convenient way are described.

Abstract: Disclosed herein is a novel chimeric antigen receptor and use thereof. The novel chimeric antigen receptor consists of a signal peptide, an antigen binding domain, a transmembrane region, and an intracellular signal domain, and comprises a 4-1BB signal peptide and/or a 4-1BB molecular transmembrane region. Nucleic acid sequences of various chimeric antigen receptors are separated and purified and provided is a chimeric antigen receptor and a CAR-T cell which are specific for a CD19 malignant tumor antigen. In the malignant tumor killing test of hematological cell lines, the ability of immune cells to target and recognize tumor cells is significantly enhanced, and the killing activity against tumor cells is also enhanced.

Abstract: A preparation method and use of an artificial exosome complex are provided. Raw materials for preparing the artificial exosome complex include linear polyethyleneimine (PEI) and hyaluronic acid (HA). The linear PEI can be obtained through the HA-PEI preparation process of the present disclosure, and most PEI is branched currently. According to the HA-PEI preparation method, HA-PEI with a specific molecular weight can be quantitatively prepared. The preparation process involves controllable operation steps, and thus can be used for large-scale industrial production. The prepared HA-PEI can promote the absorption of an encapsulated active substance by deep skin cells through the ligand activity of HA and the membrane permeability of linear PEI.

Abstract: A recombinant vector having an expression cassette is provided which comprises a modified human low density lipoprotein receptor (hLDLR) gene is provided, wherein said hLDLR gene encodes a modified hLDLR comprising (a) one or more of the following amino acid substitutions: L318H, N295D, H306D, V307D, N309A, D310N, L318H, and/or L318D; or (b) an amino acid substitution of any of (a) in combination with one or more of the following amino acid substitutions: K796, K809R and/or C818A. Also provided are pharmaceutical compositions containing this vector and uses therefor in lowering cholesterol and/or treating familial hypercholesterolemia.

Abstract: The present disclosure provides engineered Class 1 Type I CRISPR-Cas (Cascade) systems that comprise multi-protein effector complexes, nucleoprotein complexes comprising Type I CRISPR-Cas subunit proteins and nucleic acid guides, polynucleotides encoding Type I CRISPR-Cas subunit proteins, and guide polynucleotides. Also, disclosed are methods for making and using the engineered Class 1 Type I CRISPR-Cas systems of the present invention.

Abstract: The invention provides specific transgenic soybean plants, plant material and seeds, characterized in that these products harbor a specific nematode resistance and herbicide tolerance transformation event at a specific location in the soybean genome. Tools are also provided which allow rapid and unequivocal identification of the event in biological samples.

Abstract: Compositions and methods for introducing double-stranded breaks within the SERPINA1 gene are provided. Compositions and methods for reducing and eliminating mutant forms of ?1-antitrypsin (AAT), such as seen in subjects having ?1-antitrypsin deficiency (AATD), are provided.

Abstract: This disclosure pertains to a novel platform for genetic engineering of chloroplasts. The disclosure provides episomal DNA vectors containing a chloroplast origin of replication. These vectors remain extra-plastomic and sustainably and autonomously replicate in chloroplasts of the plant cells transformed with the vectors and in the plants regenerated from the transformed plant cells. The episomal DNA vectors do not contain any sequence that shares sequence homology with the plastome DNA and, thus, do not get integrated into the plastome DNA. The vectors can also comprise one or more genes of interest that confer desirable characteristics to the transformed plant cells. The disclosure also provides methods of transforming plant cells with the episomal DNA vectors and regenerating from the transformed plant cells plants having desirable characteristics.

Abstract: The ?-glucan-binding protein contains an amino acid sequence represented by SEQ ID NO: 1.

Abstract: Provided herein are methods for preparing a sequencing library that includes nucleic acids from a plurality of single cells. In one embodiment, the sequencing library includes whole genome nucleic acids from the plurality of single cells. In one embodiment, the method includes generating nucleosome-depleted nuclei by chemical treatment while maintaining integrity of the nuclei. Also provided herein are compositions, such as compositions that include chemically treated nucleosome-depleted isolated nuclei.

Abstract: The present disclosure provides improved genome editing compositions and methods for editing a TGF?R2 gene. The disclosure further provides genome edited cells for the prevention, treatment, or amelioration of at least one symptom of, a cancer, an infectious disease, an autoimmune disease, an inflammatory disease, or an immunodeficiency.

Abstract: The present invention is directed to an artificial nucleic acid and to polypeptides suitable for use in treatment or prophylaxis of an infection with Henipavirus, particularly Hendra virus and/or Nipah virus or a disorder related to such an infection. In particular, the present invention concerns a Hendra virus and/or Nipah virus vaccine. The present invention is directed to an artificial nucleic acid, polypeptides, compositions and vaccines comprising the artificial nucleic acid or the polypeptides. The invention further concerns a method of treating or preventing a disorder or a disease, first and second medical uses of the artificial nucleic acid, polypeptides, compositions and vaccines. Further, the invention is directed to a kit, particularly to a kit of parts, comprising the artificial nucleic acid, polypeptides, compositions and vaccines.

Abstract: Improved methods and means are provided to modify in a targeted manner the genome of a plant cell or plant at a predefined site via bacterial transformation.

Abstract: The present disclosure relates to materials and methods for spatially analyzing nucleic acids that have been fragmented with a transposase enzyme, alone or in combination with other types of analytes.

Abstract: The specificity of CD4+ TH responses of German cockroach (Bla g) antigens, and whether differences exist in magnitude or functionality as a function of disease severity, is disclosed. Also disclosed are novel German cockroach allergens and epitopes.

Abstract: Adeno-associated virus rh.10 sequences, vectors containing same, and methods of use are provided.

Abstract: The present invention concerns the use of antisense oligonucleotides (AON) capable of inhibiting expression of dynamin 2, advantageously human dynamin 2, for use in the treatment of Charcot-Marie-Tooth disease (CMT) and centronuclear myopathies (CNM).

Abstract: The present disclosure provides an innovative method for improving the enzyme activity of an NMN biosynthetic enzyme Nampt, and relates to the technical field of genetic engineering. A mutant protein of the present disclosure is obtained by firstly analyzing a target protein Nampt using two softwares FoldX and DeepDDG, and then predicting multiple key sites influencing the enzyme functions and finally performing the semi-rational design of the enzyme. In the examples of the present disclosure, 10 mutant strains are constructed using the designed primers according to the principle of point mutation, and 8 of the mutants have higher activity than a wild-type strain, in which the NMN yield of the mutant Nampt-V365L is increased by 62%, and the NMN yields of the mutants Nampt-S248A, Nampt-N164L, Nampt-S382M, Nampt-A245T and Nampt-A208G are increased by 34%, 27%, 27%, 22% and 17% respectively.

Abstract: Recombinant cells and methods therefor are contemplated that allow for rapid and high titer production of recombinant viruses, and especially replication deficient Ad5 virus. In some preferred aspects, the host cell is modified to produce an inhibitor that reduces or eliminates the expression of a therapeutic protein encoded in the virus, while in other aspects, the virus includes a gene that directly or indirectly reduces or eliminates the expression of a therapeutic protein encoded in the virus. Most preferably, shRNA encoded by the host cell will reduce or suppress expression of a payload gene encoded in the recombinant virus.

Abstract: A highly glycosylated human blood-clotting factor VIII (FVIII) fusion protein, and a manufacturing method and application of same. The fusion protein comprises, from the N-terminus to the C-terminus, a human (FVIII), a flexible peptide connector, at least one rigid unit of a human chorionic gonadotropin ?-subunit carboxyl terminal peptide, and a half-life extending portion (preferentially selected from a human IgG Fc variant). The fusion protein has a similar level of biological activity as a recombinant (FVIII) and an extended in vivo half-life, thereby improving pharmacokinetics and drug efficacy.

Abstract: The present invention relates to an oncolytic virus which is, or is derived from, a clinical isolate which has been selected by comparing the abilities of a panel of three or more clinical isolates of the same viral species to kill tumor cells of two or more tumor cell lines in vitro and selecting a clinical isolate which is capable of killing cells of two or more tumor cell lines more rapidly and/or at a lower dose in vitro than one or more of the other clinical isolates in the panel.

Abstract: The present disclosure relates to a novel method of producing vanillin and/or derivatives thereof by applying improved biocatalysts. Also provided herein are expression systems for preparing said improved biocatalysts. Moreover provided herewith are novel enzyme mutants, corresponding coding sequences and vectors applicable in the biochemical production of vanillin. The present disclosure further provides recombinant host cells or organisms genetically modified for improved functional expression of biocatalysts, as well as recombinant host cells or organisms useful to produce vanillin.

Abstract: The invention relates to methods and compositions for identifying and selecting maize plants with enhanced resistance to northern leaf blight. Maize plants generated by the methods of the invention are also a feature of the invention.

Abstract: Provided herein are methods for using compositions that include a fusion protein having a YscF protein domain, a mature F1 protein domain, and a LcrV protein domain. In one embodiment the composition is used to confer immunity to plague, such as pneumonic plague, caused by Yersinia pestis. In one embodiment, the composition is administered to a mucosal surface, such as by an intranasal route. In one embodiment, the administration to a mucosal surface includes a vector that has a polynucleotide encoding a fusion protein, where the fusion protein includes a YscF protein domain, a mature F1 protein domain, and a LcrV protein domain. The administration is followed by a second administration by a different route, such as an intramuscular route. The second administration includes a fusion protein having the same three domains, and in one embodiment the fusion protein is the same one administered to a mucosal surface.