Abstract: A biodegradable cationic polymer is disclosed, comprising first repeat units derived from a first cyclic carbonyl monomer by ring-opening polymerization, wherein more than 0% of the first repeat units comprise a side chain moiety comprising a quaternary amine group; a subunit derived from a monomeric diol initiator for the ring-opening polymerization; and an optional endcap group. The biodegradable cationic polymers have low cytotoxicity and form complexes with biologically active materials useful in gene therapeutics and drug delivery.

Abstract: Disclosed herein are methods for contacting in a closed container a host liquid including target cells, microbubble reagents comprising gas-core lipid-shelled microbubbles, and one or more antibodies or other ligands that bind to cell surface molecules on the target cells, wherein the one or more antibodies or other ligands are bound to the target cells or the microbubbles, wherein the contacting under conditions to produce target cells linked to microbubbles via the one or more antibodies or other ligands and activating the target cells to generate activated target cells.

Abstract: A compound comprising formula (I): wherein R1 is an akylamino group or a group containing at least one aromatic group; R2 and R3 are independently an aliphatic group or hydrophobic group; R4 and R5 are independently H, a substituted or unsubstituted akyl group, an akenyl group, an acyl group, an aromatic group, polymer, a targeting group, or a detectable moiety; a, b, c, and d are independently an integer from 1 to 10; and pharmaceutically acceptable salts thereof.

Abstract: Provided, in part, is a composition comprising one or more chemical entities of formula I, each of which is a compound of formula I: a pharmaceutically acceptable salt thereof, a solvate thereof, or a solvate of a pharmaceutically acceptable salt thereof, the composition characterized in that greater than a first threshold amount of the total amount of chemical entities of formula I in the composition: are chemical entities of formula I.a, wherein the first threshold amount is 50%; or are chemical entities of formula I.b.1, wherein the first threshold amount is 25%; or are chemical entities of formula I.b.2, wherein the first threshold amount is 25%, wherein the chemical entities of formula I.a, I.b.1, and I.b.2, are described herein, and methods of using such compositions, for example, for the delivery of a polynucleotide in vivo.

Abstract: Materials and methods for treating a patient with hereditary hemochromatosis (HHC), both ex vivo and in vivo, and materials and methods for modulating the expression, function, or activity of a haemochromatosis (HFE) gene in a cell by genome editing.

Abstract: The present invention provides polynucleotide vectors for high expression of heterologous genes. Some vectors further comprise novel transposons and transposases that further improve expression. Further disclosed are vectors that can be used in a gene transfer system for stably introducing nucleic acids into the DNA of a cell. The gene transfer systems can be used in methods, for example, gene expression, bioprocessing, gene therapy, insertional mutagenesis, or gene discovery.

Abstract: A method for preventing or treating an inflammatory disease, such as inflammatory bowel disease and Crohn disease, is presented. The method includes administering to a patient a polypeptide having the amino acid sequence of SEQ ID NO: 1, a conservative derivative or a fragment thereof, a nucleic acid sequence encoding said polypeptide, a vector comprising said nucleic acid sequence or a host cell that has been transfected, infected or transformed by said nucleic acid sequence and/or by said vector.

Abstract: The present invention relates to dry particulate encapsulation compositions comprising a water-insoluble matrix comprising at least 70% by weight of proteins, based on the total weight of the matrix and a moisture content of about 5 to 10% by weight, based on the total weight of the matrix and an encapsulate encapsulated in the matrix, wherein the matrix once wetted in a clear colorless aqueous solution or in mineral oil has a lightness value (L*) greater than about 40, a color vividness or Chroma (C*) lower than about 33 and a hue angle between about 70 and 90. The encapsulation compositions of the present invention are useful in encapsulating dyes, medications and vitamins. Fine particulate encapsulation compositions comprising natural dyes can be used in lieu of artificial lakes in confectionery, cosmetics and caplets color coatings.

Abstract: The present disclosure relates to an additive for soil conditioning and an agricultural composition comprising additive and plant growth regulator. The additive comprises a mixture of esters of fatty acids and an emulsifying agent. The additive of the present disclosure can be used in the conditioning of the soil which results in improvement in plant growth. The agricultural composition of the present disclosure can be used for improving soil condition and promoting plant growth.

Abstract: A method of curing (or at least treating) HIV, by obtaining small tissue mesenchymal stem cells (stMSC) from adipose tissue of a patient with HIV or from a syngeneic donor or an immuno-compatible donor, wherein said stMSC are i) 4-6 ?m in diameter, ii) pluripotent, and iii) have CD11b?, CD34?, CD45?, CD 29, CD 49, Oct 4 and SSEA 4 surface markers when harvested. Preferred sources are autologous adipose tissue. Next those stMSCs are gene edited to provide CCR5? stMSCs and optionally amplified by growth in culture and/or by cell selection for gene edited stMSCs and then re-introducing into the patient. Preferably, the CCR5? stMSCs will re-populate the patient with stem cells that can no longer act as latent HIV reservoirs and can differentiate into immune cells that are HIV resistant. If desired, the patient can be first treated, e.g., by radiation, to destroy the bone marrow cells before the re-introduction.

Abstract: The present invention provides nucleic acids, vectors, host cells, methods and compositions to confer and/or augment immune responses mediated by cellular immunotherapy, such as by adoptively transferring CD8+ central memory T cells or combinations of central memory T cells with CD4+ T cells that are genetically modified to express a chimeric receptor. In embodiments the genetically modified host cell comprises a nucleic acid comprising a polynucleotide coding for a ligand binding domain, a polynucleotide comprising a customized spacer region, a polynucleotide comprising a transmembrane domain, and a polynucleotide comprising an intracellular signaling domain. It has been surprisingly found that the length of the spacer region can affects the ability of chimeric receptor modified T cells to recognize target cells in vitro and affects in vivo efficacy of the chimeric receptor modified T cells. Pharmaceutical formulations produced by the method, and methods of using the same, are also described.

Abstract: Disclosed is an oil-in-water anionic emulsion for eyelashes, and methods for its use, comprising a continuous phase comprising 35 to 55% by weight of water based on the total weight of the emulsion, a discontinuous phase comprising 15 to 25% by weight of a combination of waxes, wherein the combination of waxes comprises paraffin wax, carnauba wax, beeswax, and candelilla wax based on the total weight of the emulsion, and 5 to 15% by weight of the emulsion of an anionic surfactant system comprising stearic acid, palmitic acid, myristic acid, polyethylene glycol-40 (PEG-40) stearate, and stearyl stearate, wherein the oil-in-water anionic emulsion is capable of thickening the appearance of eyelashes.

Abstract: The disclosure features novel lipids and compositions involving the same. Nanoparticle compositions include a novel lipid as well as additional lipids such as phospholipids, structural lipids, and PEG lipids. Nanoparticle compositions further including therapeutic and/or prophylactics such as RNA are useful in the delivery of therapeutic and/or prophylactics to mammalian cells or organs to, for example, regulate polypeptide, protein, or gene expression.

Abstract: The method of producing eggshell-derived nanoparticles may include steps of adding eggshell powder to methanol to form a solution; adding the solution dropwise to boiling water under ultrasonic conditions; incubating the resulting solution under continuous stirring at 200-800 rpm; and drying the resulting solution to obtain the eggshell-derived nanoparticles. The method produces nanoparticles of between 5 and 100 nm. Cytotoxicity testing shows that the nanoparticles exhibit anticancer activity against human breast cancer and lung cancer cell lines.

Abstract: Provided herein are compositions and methods for treating craniosynostosis. In particular, provided herein are methods of treating and preventing craniosynostosis by administering an isolated TNAP polypeptide or a nucleic acid molecule that encodes a TNAP polypeptide.

Abstract: The present disclosure is in the field of genome engineering, particularly targeted modification of the genome of a hematopoietic cell.

Abstract: A method of decreasing cytokine production and release is disclosed. In one embodiment, the method comprises the step of providing cytotoxic cells to a subject wherein the cells are preferably natural killer cells or T lymphocytes and are genetically modified to express a chimeric antigen receptor comprising a first element that is an extracellular antigen receptor and a second intracellular element that is a signaling moiety comprising altered ADAP-dependent or Fyn-dependent signaling such that downstream signaling causing cytokine release is decreased. Specific modifications of CD137 and NKG2D cytoplasmic tails are described. Additionally, methods to develop and screen drug compounds capable of compromising the binding between ADAP and Fyn and disrupting the downstream release of cytokines are described.

Abstract: The present invention provides nucleic acids, vectors, host cells, methods and compositions to confer and/or augment immune responses mediated by cellular immunotherapy, such as by adoptively transferring CD8+ central memory T cells or combinations of central memory T cells with CD4+ T cells that are genetically modified to express a chimeric receptor. In embodiments the genetically modified host cell comprises a nucleic acid comprising a polynucleotide coding for a ligand binding domain, a polynucleotide comprising a customized spacer region, a polynucleotide comprising a transmembrane domain, and a polynucleotide comprising an intracellular signaling domain. It has been surprisingly found that the length of the spacer region can affects the ability of chimeric receptor modified T cells to recognize target cells in vitro and affects in vivo efficacy of the chimeric receptor modified T cells. Pharmaceutical formulations produced by the method, and methods of using the same, are also described.

Abstract: The present invention provides nucleic acids, vectors, host cells, methods and compositions to confer and/or augment immune responses mediated by cellular immunotherapy, such as by adoptively transferring CD8+ central memory T cells or combinations of central memory T cells with CD4+ T cells that are genetically modified to express a chimeric receptor. In embodiments the genetically modified host cell comprises a nucleic acid comprising a polynucleotide coding for a ligand binding domain, a polynucleotide comprising a customized spacer region, a polynucleotide comprising a transmembrane domain, and a polynucleotide comprising an intracellular signaling domain. It has been surprisingly found that the length of the spacer region can affects the ability of chimeric receptor modified T cells to recognize target cells in vitro and affects in vivo efficacy of the chimeric receptor modified T cells. Pharmaceutical formulations produced by the method, and methods of using the same, are also described.

Abstract: The present invention provides diacylhydrazine ligands and chiral diacylhydrazine ligands for use with ecdysone receptor-based inducible gene expression systems. Thus, the present invention is useful for applications such as gene therapy, large scale production of proteins and antibodies, cell-based screening assays, functional genomics, proteomics, metabolomics, and regulation of traits in transgenic organisms, where control of gene expression levels is desirable. An advantage of the present invention is that it provides a means to regulate gene expression and to tailor expression levels to suit the user's requirements.