Abstract: Exemplary embodiments of the disclosure may be drawn to a device having an inlet and a chamber. Immobilized lipase, immobilized protease, and immobilized amylase may be contained within the chamber. The device may also include an outlet, wherein a flow path extends from the inlet, through the chamber, and to the outlet.

Abstract: Disclosed are adenosine deaminases, base editors comprising the adenosine deaminases and complexes comprising the base editors. The adenosine deaminases and the base editors exhibited superior adenine editing effects and achieved A·T base pair to G·C base pair transformation at DNA level.

Abstract: Compositions and methods are provided for novel Cas9 orthologs, including, but not limiting to, novel guide polynucleotide/Cas9 endonucleases complexes, single or dual guide RNAs, guide RNA elements, and Cas9 endonucleases. The present disclosure also describes methods for creating a double strand break in a target polynucleotide, methods for genome modification of a target sequence under various in vivo and in vitro conditions, in the genome of a cell, for gene editing, and for inserting a polynucleotide of interest into the genome of a cell. Also provided are nucleic acid constructs and cells having a modified target site or altered polynucleotide of interest produced by the methods described herein.

Abstract: The present application provides materials and methods for treating a patient with Duchenne Muscular Dystrophy (DMD) both ex vivo and in vivo. In addition, the present application provides materials and methods for editing a dystrophin gene in a cell by genome editing.

Abstract: Modular dendrimers with cationic groups and lipophilic groups are provided herein. In some aspects, the dendrimers provided herein may be formulated in compositions which contain a nucleic acid and one or more helper excipients. In some aspects, these compositions may also be used to treat diseases or disorders with a therapeutic nucleic acid.

Abstract: Modular dendrimers with cationic groups and lipophilic groups are provided herein. In some aspects, the dendrimers provided herein may be formulated in compositions which contain a nucleic acid and one or more helper excipients. In some aspects, these compositions may also be used to treat diseases or disorders with a therapeutic nucleic acid.

Abstract: Provided herein are methods and compositions for performing highly sensitive in vitro assays to define substrate preferences and off-target sites of nucleic-acid binding, modifying, and cleaving agents.

Abstract: A method for modifying double stranded DNA (dsDNA) employing an RNA guided DNA endonuclease to generate two double strand breaks in the dsDNA molecule to be modified, and replacement of the sequence positioned between the double strand breaks with a substitute DNA sequence using the non-homologous end joining (NHEJ) pathway, and corresponding kits and compositions for modifying double stranded DNA molecules.

Abstract: A composition comprising at least one AAV vector formulated for central nervous system delivery is described. The composition comprises at least one expression cassette which contains sequences encoding an anti-neoplastic immunoglobulin construct for delivery to the brain operably linked to expression control sequences therefor and a pharmaceutically acceptable carrier. The anti-neoplastic immunoglobulin construct may be an immunoglobulin modified to have decreased or no measurable affinity for neonatal Fc receptor (FcRn). Also provided are methods of using these constructs in preparing pharmaceutical compositions and uses thereof in anti-neoplastic regimens, particularly for primary and/or metastatic cancers of the brain.

Abstract: Provided herein are systems, compositions, and methods of introducing protective and/or loss-of-function variants of CCR5 and CCR2. Variants may be introduced using a CRISPR/Cas9-based nucleobase editor or other guide nucleotide sequence-programmable DNA binding protein domain-based fusion protein described herein. Further provided herein are compositions and methods of preventing and treating conditions related to HIV infection and progression as well as to AIDS.

Abstract: The invention relates to improved alkaline phosphatases, pharmaceutical compositions comprising improved alkaline phosphatases and the use of improved alkaline phosphatases for preventing, treating or curing diseases.

Abstract: The invention relates to the use of deoxyribonuclease (DNase) enzyme for inhibiting progression and for prevention and treatment of neurodegeneration.

Abstract: The present invention relates to a novel cancer-associated biomarker and different applications and uses thereof. More specifically, the invention relates to a novel splice variant of CIP2A denoted as NOCIVA, as well as binding bodies such as probes, amplification primers, and antibodies specific for the same. Also provided are various methods for detecting and prognosing cancer on the basis of said splice variant, and a kit for use in said methods.

Abstract: The present invention relates to, inter alia, compositions and methods, including therapeutic alkaline phosphatases that find use in the treatment or prevention of the effects of radiation exposure and/or chemotherapy.

Abstract: A method for purification of a sulfatase using metal chelating chromatography without using tags such as His-tag, etc. is disclosed. An embodiment provides a method for purifying a sulfatase including the steps of: (a) providing a sulfatase-containing solution comprising one or a plurality of impurities; (b) performing a first chromatographic separation of the sulfatase-containing solution using a metal affinity chromatography resin; (c) performing a second chromatographic separation using a cation exchange chromatography resin; and (d) performing a final chromatographic separation using an anion exchange chromatography resin, wherein the impurities are removed thereby.

Abstract: Disclosed herein are methods for treating a subject with an alkaline phosphatase deficiency, further comprising monitoring additional analytes, e.g., calcium, parathyroid hormone and/or vitamin D, with treatment modifications as indicated by the levels, e.g., serum levels, of the additional analytes.

Abstract: The present invention concerns a postbiotic composition comprising at least one postbiotic and at least one bacteriocin and/or endolysin, preferably formulated, and a postbiotic composition comprising at least one postbiotic and at least one bacteriocin and/or endolysin for use as a medicament, wherein said postbiotic is preferably a microbial lysate, preferably obtained from microorganisms heterologously expressing said at least one bacteriocin and/or endolysin and wherein said at least one postbiotic and said at least one bacteriocin and/or endolysin have a synergistic effect in the therapeutic treatment.

Abstract: The present invention relates to polypeptides, nucleotides encoding the polypeptide, as well as methods of producing the polypeptides. The present invention also relates to detergent composition comprising polypeptides, a laundering method and the use of polypeptides.

Abstract: The present invention provides, among other things, improved methods for purifying I2S protein produced recombinantly for enzyme replacement therapy. The present invention is, in part, based on the surprising discovery that recombinant I2S protein can be purified from unprocessed biological materials, such as, I2S-containing cell culture medium, using a process involving as few as four chromatography columns.

Abstract: Disclosed herein is an endotoxin-free asparaginase enzyme. Also disclosed are methods of using the disclosed enzyme to treat subjects with a disease treatable by depletion of asparagine. For example, the disclosed endotoxin-free asparaginase enzyme is useful in the treatment or the manufacture of a medicament for use in the treatment of acute lymphoblastic leukemia (ALL) in both adults and children, as well as other conditions where asparagine depletion is expected to have a useful effect.

Abstract: The present disclosure relates to methods for using one or more polypeptides with phytase activity in grain processing, ethanol, and biofuel production.

Abstract: The present invention relates to chimeric neurotoxins with enhanced properties and their use in therapy.

Abstract: The invention relates to methods of treating or preventing organophosphate poisoning in a subject in need thereof, comprising administering to the subject isoflurane, enflurane, halothane, sevoflurane, desflurane, xenon or argon in a therapeutically effective amount.

Abstract: The present disclosure provides compositions, methods and kits for the treatment of cancer. Particularly, the present disclosure provides synergistic compositions comprising at least one cytotoxic ribonuclease and at least one MAPK-pathway inhibitor.

Abstract: PNPase inhibitors (such as 8-substituted guanine and/or 8-substituted guanosine) and/or PNPase purine nucleoside substrates (such as guanosine and/or inosine) can also be used in methods of treating SCD or reducing sickling of red blood cells (RBCs). Herein, 8-substituted guanine and/or 8-substituted guanosine compounds are used in methods to treat hypertension (such as systemic or pulmonary hypertension), stroke, diabetes, and/or patients in need of a diuretic. In addition, 8-substituted guanine and/or 8-substituted guanosine compounds are included in a beverage composition. Further, purine nucleoside phosphorylase (PNPase) inhibitors (such as 8-substituted guanine and/or 8-substituted guanosine) and/or PNPase purine nucleoside substrates (such as guanosine and/or inosine) can be used in methods of treating or reducing the risk of pulmonary hypertension (PH), such as PH associated with HIV or sickle cell disease (SCD).

Abstract: Provided are mutant cocaine esterase polypeptides and PEGylated formulations thereof.

Abstract: The present invention relates to prevention of congenital deformations. The invention further relates to cancer inhibition and prevention. The invention further relates to methods and compositions to modulate, antagonize, or agonize disparate signaling pathways that may converge to regulate patterning events and gene expression during prenatal development, post-natal development, and during development in the adult organism. The invention also relates to activators or deactivators of pyruvate kinase M2 (PKM2) for the treatment, prevention, or amelioration of diseases related to PKM2 function.

Abstract: The aim of the present invention is to provide a fluid composition that can form a coating, said composition being characterized in that it contains, at least in a solvent medium, an effective amount of at least one natural virucide selected from among lauric acid, monolaurin, lactoferrin and essential oils having antiviral activity and/or one of the precursors thereof, said composition having a viscosity of 30 mPa·s to 40 Pa·s at room temperature and pressure.

Abstract: The invention relates to expressing proteins in the axons of mammalian neurons. The invention provides nucleic acids that can be used to express a selected polypeptide in neuronal axons, viruses that can be used deliver nucleic acids of the invention into neuronal axons, as well as methods for doing so. Thus, the invention provides pharmaceutical compositions comprising viruses of the invention, as well as their use in methods of treating injured axons or conditions associated with aberrant axon growth or function.

Abstract: This invention provides compositions of active highly phosphorylated lysosomal sulfatase enzymes, their pharmaceutical compositions, methods of producing and purifying such lysosomal sulfatase enzymes and compositions and their use in the diagnosis, prophylaxis, or treatment of diseases and conditions, including particularly lysosomal storage diseases that are caused by, or associated with, a deficiency in the lysosomal sulfatase enzyme.

Abstract: The present invention provides a method for treating and/or ameliorating a disorder of the lower urinary tract, comprising administering a composition comprising a glucagon like peptide 2 (GLP-2) receptor agonist to a patient in need thereof in an amount sufficient to treat and/or ameliorate the disorder of the lower urinary tract.

Abstract: The present invention relates to an improved method of providing photoreceptor function to a cell, for example for use in the treatment of retinal degeneration. The present invention also relates to compositions and kits, in particular for use in such methods.

Abstract: Composition and methods are provided for the specific manipulation of protein tyrosine phosphatase (PTP) activity, including without limitation manipulation of protein tyrosine phosphatase receptor type gamma (PTPRG). The modulation of PTP activity can be performed in vitro or in vivo, and is useful for therapeutic and research purposes. In some embodiments, an effective dose of a PTP modulator is provided to an individual for preventing or treating disease involving dysregulated tyrosine kinase activity and/or signaling mechanisms involving tyrosine phosphorylation and/or tyrosine kinase activity. In other embodiments, a PTP modulator is utilized in the analysis and screening of phosphatase pathways in a cell.

Abstract: The present disclosure provides biophotonic compositions, kits and their uses. In particular, the biophotonic compositions of the present disclosure are substantially resistant to leaching such that low amounts of chromophores present in the biophotonic composition leach out of the composition. The biophotonic compositions and their uses are useful for promoting repair of non-healing wounds.

Abstract: The present invention provides a method of treating or preventing immunoinflammatory, vascular, thrombotic or ischemic disorders in a subject, the method comprises administering to the subject an agent which dissipates nucleotide phosphates or generates a product which stimulates adenosine receptors. The present invention also provides a method of treating or preventing immunoinflammatory, thrombotic or ischemic disorders in a subject by inhibiting leukocyte infiltration into a site which comprises administering to the subject an effective amount a described agent. Agents described for use in the methods of the invention include CD73, a fragment a mutant, or a modified form thereof.

Abstract: The present invention comprises a method to diminish and/or eliminate atherosclerotic plaques, in mammals, through direct and indirect treatment of these plaques, in situ, using suitable substances which are capable of lipid removal, primarily through hydrolysis, either by a catalytic or stoichiometric process, wherein the substance targets receptors in and/or on the cell which lead to uptake into the lysosome. Such substances used to diminish and/or eliminate atherosclerotic plaques are generally comprised of lipid hydrolyzing proteins and/or polypeptides.

Abstract: The present invention relates to immunogenic polypeptide fragments of a human Arginase protein. The fragments are in particular useful for the treatment or prevention of cancer.

Abstract: An interferometric scattering microscope is adapted by performing spatial filtering of output light, which comprises both light scattered from a sample location and illuminating light reflected from the sample location, prior to detection of the output light. The spatial filtering passes the reflected illumination light but with a reduction in intensity that is greater within a predetermined numerical aperture than at larger numerical apertures. This enhances the imaging contrast for coherent illumination, particularly for objects that are weak scatterers.

Abstract: The presently-disclosed subject matter includes isolated polypeptides that comprise a butyrylcholinestrase (BChE) polypeptide and a second polypeptide. The BChE polypeptide as well as the second polypeptide can be variants and/or fragments thereof. The presently-disclosed subject matter also includes a pharmaceutical composition that comprises the present isolated polypeptide and a suitable pharmaceutical carrier. Further still, methods are provided for treating cocaine-induced conditions, and comprise administering the isolated polypeptide and/or pharmaceutical compositions thereof to an individual.

Abstract: The present invention is directed to novel methods comprising the isolation of viable fetal progenitor cells from organs of aborted fetus. The invention comprises the use of natural proteolytic, collagenolytic and fibrinolytic activity of autologous abortive placenta tissue extract.

Abstract: Provided herein are systems, compositions, and methods of introducing protective and/or loss-of-function variants of CCR5 and CCR2. Variants may be introduced using a CRISPR/Cas9-based nucleobase editor or other guide nucleotide sequence-programmable DNA binding protein domain-based fusion protein described herein. Further provided herein are compositions and methods of preventing and treating conditions related to HIV infection and progression as well as to AIDS.

Abstract: The present invention relates to animal protein-free cell culture media comprising polyamines and a plant- and/or yeast-derived hydrolysate. The invention also relates to animal protein-free culturing processes, wherein cells can be cultivated, propagated and passaged without adding supplementary animal proteins in the culture medium. These processes are useful in cultivating cells, such as recombinant cells or cells infected with a virus, and for producing biological products by cell culture processes.

Abstract: Methods and compositions to restore function to acidified T cells are provided. The methods comprise administering urease to the T cells. Compositions comprise urease.

Abstract: The present invention discloses a lysin that is capable of killing Staphylococcus and the use thereof, belonging to the field of biological agents. The present invention discloses the amino acid sequence and the encoding gene sequence of the lysin. This lysin keeps active in a wide range of pH. It has lytic activity against Staphylococcus in pH 4-11. The recombinant protease constructed by the encoding gene can be solubly expressed in E. coli strain BL21 (DE3). The lysin can be used to effectively kill multiple species Staphylococcus in vitro, including methicillin sensitive Staphylococcus aureus (MSSA) and methicillin resistant Staphylococcus aureus (MRSA) isolated in clinics. This lysin can be used as an antibiotic for the treatment of staphylococcal infections in vivo. This lysin is also able to rapidly lyse staphylococcal cell wall; as a result, intracellular substances such as ATP and DNA are released. Those released substances can be used to detect the type of Staphylococcus.

Abstract: This invention provides compositions of active highly phosphorylated lysosomal sulfatase enzymes, their pharmaceutical compositions, methods of producing and purifying such lysosomal sulfatase enzymes and compositions and their use in the diagnosis, prophylaxis, or treatment of diseases and conditions, including particularly lysosomal storage diseases that are caused by, or associated with, a deficiency in the lysosomal sulfatase enzyme.

Abstract: The present invention discloses a composition for biomass hydrolysis. The components of the composition may be obtained by biological or synthetic means. Synthetically, the components of the composition of the present invention may be obtained by amino acid synthesis or may be procured commercially.

Abstract: The present invention provides compositions of recombinant human lysosomal acid lipase having particular glycosylation patterns for internalization into target cells, a vector containing the nucleic acid encoding human lysosomal acid lipase, a host cell transformed with the vector, pharmaceutical compositions comprising the recombinant human lysosomal acid lipase and method of treating conditions associated with lysosomal acid lipase deficiency.

Abstract: The present invention provides, among other things, improved methods for purifying I2S protein produced recombinantly for enzyme replacement therapy. The present invention is, in part, based on the surprising discovery that recombinant I2S protein can be purified from unprocessed biological materials, such as, I2S-containing cell culture medium, using a process involving as few as four chromatography columns.

Abstract: The invention provides hydrolases, polynucleotides encoding them, and methods of making and using these polynucleotides and polypeptides. In one aspect, the invention is directed to polypeptides, e.g., enzymes, having a hydrolase activity, e.g., an esterase, acylase, lipase, phospholipase (e.g., phospholipase A, B, C and D activity, patatin activity, lipid acyl hydrolase (LAH) activity) or protease activity, including thermostable and thermotolerant hydrolase activity, and polynucleotides encoding these enzymes, and making and using these polynucleotides and polypeptides. The hydrolase activities of the polypeptides and peptides of the invention include esterase activity, lipase activity (hydrolysis of lipids), acidolysis reactions (to replace an esterified fatty acid with a free fatty acid), transesterification reactions (exchange of fatty acids between triglycerides), ester synthesis, ester interchange reactions, phospholipase activity and protease activity (hydrolysis of peptide bonds).

Abstract: Viral infections of the eye, and particularly viral infections in the Herpesviridae and Adenoviridae families, can be treated by administration of a pharmaceutical made up of an enzymatically active ribonuclease and a vehicle. Advantageously, the enzymatically active ribonuclease is ranpirnase, the '805 variant, rAmphinase 2, and Amphinase 2, and the vehicle is an aqueous solution.