Abstract: The present invention is in the field of drug delivery, and specifically, cationic liposome-based drug delivery. In embodiments, this invention provides methods of making ligand-targeted (e.g., antibody- or antibody fragment-targeted) liposomes useful for the delivery of liposomes to tumors, including brain tumors. In embodiments, the liposomes deliver temozolomide across the blood-brain barrier for treatment of primary or metastatic brain tumors. Additional cancers that can be treated with the liposomes include neuroendocrine tumors, melanoma, prostate, head and neck, ovarian, lung, liver, kidney, breast, urogenital, gastric, colorectal, cervical, vaginal, angiosarcoma, liposarcoma, rhabdomyosarcoma, choriocarcinoma, pancreatic, retinoblastoma and other types of cancer. In another embodiment the liposomes deliver melphalan for the treatment of multiple myeloma, other tumors of the blood or other solid tumors.

Abstract: The present disclosure details various lipids, compositions, and/or methods of optimized systems and delivery vehicles for the delivery of nucleic acid sequences, polypeptides or peptides for use in vaccinating against infectious agents.

Abstract: The disclosure features novel methods of producing nucleic acid lipid nanoparticle (LNP) compositions employing a modifying agent after formation of a precursor nucleic acid lipid nanoparticle, the produced compositions thereof, and methods involving the nucleic acid lipid nanoparticles useful in the delivery of therapeutics and/or prophylactics, such as a nucleic acid, to mammalian cells or organs to, for example, to regulate polypeptide, protein, or gene expression.

Abstract: An object of the present invention is to provide a liposome composition and a pharmaceutical composition, which exhibit a high AUC. Provided are a liposome composition including a hydrophilic polymer-modified diacylphosphatidylethanolamine, a dihydrosphingomyelin, and cholesterols as components of a liposome membrane, in which the liposome composition encapsulates a drug, an inner water phase thereof contains ammonium sulfate, and a molar ratio of sulfate ions in the inner water phase to the drug in an entire water phase is 0.36 or more; and a pharmaceutical composition including the liposome composition.

Abstract: The invention concerns a synthetic compound for enabling transfection of eukaryotic cells by means of an amphiphilic block copolymer which synthetic compound comprises a peptide residue having at least one targeting sequence and a nucleic acid binding sequence which nucleic acid binding sequence comprises or consists of at least four consecutive amino acid residues which are positively charged at pH 7.4, wherein the targeting sequence is any sequence that causes a direction of the sequence to a position or a structure inside or on the surface of eukaryotic cells, characterized in that the synthetic compound further comprises a hydrophobic moiety covalently linked to the peptide residue, wherein the hydrophobic moiety comprises or consists of lipoic acid residue, lipoamide residue, a tetradecyl residue, a cholesteryl residue, or a further peptide residue having a sequence with more than 40% amino acid residues with hydrophobic side chains.

Abstract: Aspects of the disclosure relate to nucleic acid vaccines. The vaccines include at least one RNA polynucleotides having an open reading frame encoding at least one varicella zoster virus (VZV) antigen. Methods for preparing and using such vaccines are also described.

Abstract: The present invention provides methods for de-differentiating somatic cells into stem-like cells without generating embryos or fetuses. More specifically, the present invention provides methods for effecting the de-differentiation of somatic cells to cells having stem cell characteristics, in particular pluripotency, by introducing RNA encoding factors inducing the de-differentiation of somatic cells into the somatic cells and culturing the somatic cells allowing the cells to de-differentiate.

Abstract: Provided herein are methods, compounds, and compositions for reducing expression of an ANGPTL3 mRNA and protein in an animal. Also provided herein are methods, compounds, and compositions for reducing lipids and/or glucose in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate any one or more of cardiovascular disease and/or metabolic disease, or a symptom thereof, in an individual in need thereof.

Abstract: Poly(amine-co-ester-co-ortho ester) polymers, methods of forming active agent-load nanoparticles therefrom, and methods of using the nanoparticles for drug delivery are disclosed. The nanoparticles can be coated with an agent that reduces surface charge, an agent that increases cell-specific targeting, or a combination thereof. Typically, the loaded nanoparticles are less toxic, more efficient at drug delivery, or a combination thereof compared to a control or other transfection reagents.

Abstract: The present disclosure relates to compounds and medical devices activated with a solvophobic material functionalized with a first reactive member and methods of making such compounds and devices.

Abstract: Liposomal compositions comprising of liposomes of guanidinylated cationic amphiphiles as the main lipid and cholesterol/1,2-dioleoyl-sn-glycero-3-phosphocholine (DOPC)/aminopropyl polyethyleneglycol carbamyl-distearoylphosphatidyl-ethanolamine (DSPE-peg-NH2) as co-lipids are described. These liposomal compositions containing encapsulated or electrostatically complexed therapeutic small interfering RNAs (siRNAs) against Cdc20, a key cell cycle regulator, inhibit solid tumor growth and melanoma tumor growth on lung in C57BL/6J mice.

Abstract: Disclosed herein are compositions and methods of modulating the expression of gene or the production of a protein by transfecting target cells with nucleic acids. The compositions disclosed herein demonstrate a high transfection efficacy and are capable of ameliorating diseases associated with protein or enzyme deficiencies.

Abstract: Compositions, methods, strategies, kits, and systems for the delivery of negatively charged proteins, protein complexes, and fusion proteins, using cationic polymers or lipids are provided. Delivery of proteins into cells can be effected in vivo, ex vivo, or in vitro. Proteins that can be delivered using the compositions, methods, strategies, kits, and systems provided herein include, without limitation, enzymes, transcription factors, genome editing proteins, Cas9 proteins, TALEs, TALENs, nucleases, binding proteins (e.g., ligands, receptors, antibodies, antibody fragments; nucleic acid binding proteins, etc.), structural proteins, and therapeutic proteins (e.g., tumor suppressor proteins, therapeutic enzymes, growth factors, growth factor receptors, transcription factors, proteases, etc.), as well as variants and fusions of such proteins.

Abstract: Methods of treating a wound in a subject are provided comprising administering to the subject an amount of an inhibitor of Fidgetin-like 2. Compositions and pharmaceutical compositions comprising an amount of an inhibitor of Fidgetin-like 2 are also provided. Methods are also provided for identifying an inhibitor of Fidgetin-like 2.

Abstract: A medicament for treating cystitis and a method of treatment are provided. An agent to repair the damaged glycosaminoglycan (“GAG”) layers lining the urothelium, noted in cystitis, such as pentosan polysulfate, hyaluronic acid, chondroitin, etc., is provided in a liposomal carrier. Cystitis is treated by intravesically administering to a patient a therapeutically effective dose of the medicament.

Abstract: A cell of the present invention contains a nucleic acid construct encoding a WT1 gene product or a fragment of the WT1 gene product. The nucleic acid construct contains (i) a region encoding a desired fragment of the WT1 gene product and (ii) only AUG as a functional start codon. The present invention can provide a cell into which the nucleic acid construct is introduced so that an expression level of a WT1 gene product or a fragment of the WT1 gene product is remarkably enhanced.

Abstract: A protein transduction method for efficiently delivery of exogenous proteins into mammalian cells is invented, which has the capability of targeting different cellular compartments and protection from degradation of the delivered proteins from cellular proteases. A composition for treat proteins has cation reagents, lipids and enhancers in a carrier. The method can be used in a number of ways including: production of large quantities of properly folded, post-translationally modified proteins using mammalian cell machinery, a in-cell fluorescence spectroscopy and imaging using small molecule fluorophores and a in-cell NMR spectroscopy using living mammalian cells. The method permits cell biology at atomic resolution that is physiologically and pathological relevant and permits protein therapy to treat human diseases. The method can also be used to deliver exogenous protein inside mammalian cells, wherein the exogenous proteins follow a similar secretion pathway as that of the endogenous protein.

Abstract: The present disclosure provides a method for treating and/or preventing myopia, including: administering an RNA interference (RNAi) to a subject, wherein the RNA interference is capable of counteracting another RNA interference, and the other RNA interference is an RNA interference capable of inhibiting an expression of PAX-6 gene, and the RNA interference capable of inhibiting an expression of PAX-6 gene comprises microRNA-328.

Abstract: The present invention provides a liposome based composition wherein the liposome includes a peptide conjugated thereto via a peptide bond, wherein the peptide includes a spacer amino acid and a short Apolipoprotein E recognition sequence or a short Amyloid beta recognition sequence. This invention further provides a process for making the liposome and methods of utilizing the liposome based composition for therapeutic and diagnostic purposes.

Abstract: The invention relates to a double-stranded ribonucleic acid (dsRNA) targeting a G-alpha q subunit (GNAQ) of a heterotrimeric G gene, and methods of using the dsRNA to inhibit expression of GNAQ.

Abstract: The present invention provides a recombinant bacterium and methods of using the recombinant bacterium to induce an immune response.

Abstract: Disclosed is an effective method of preparing a composition for delivering an anionic drug, the composition comprising an anionic drug as an active ingredient, a cationic lipid, and an amphiphilic block copolymer, wherein the anionic drug forms a complex with the cationic lipid, and the anionic drug/cationic lipid complex is entrapped in a micelle structure formed by the amphiphilic block copolymer.

Abstract: The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of Discs large homolog (DLG), in particular, by targeting natural antisense polynucleotides of Discs large homolog (DLG). The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorder associated with the expression of DLG.

Abstract: Methods, articles and systems that provide imagewise mapping or display of gene expression of a biosample, by contacting the biosample, such as a tissue slice or metacommunity, to a detector which captures material from the biosample and processes the captured material. In one embodiment the detector has an array of one or more capture sites at defined positions on the detector, each site carrying an immobilized capture oligonucleotide and a site-indexing oligonucleotide. The array captures mRNA from the biosample contacted thereto, and the captured mRNA is processed to form a sequenceable amount of amplified captured material which includes the site-indexing oligonucleotide, so that when sequenced, detection of the site-indexing oligonucleotide indicates the original capture location on the array, thereby mapping the sequenced material to its capture location and imaging display of gene expression distribution in the original biosample.

Abstract: Disclosed are a carrier for delivering a negatively charged drug, comprising a cationic lipid represented by formula 1, and a preparation method thereof. Also disclosed is a pharmaceutical composition comprising a negatively charged drug and a cationic lipid represented by formula 1, wherein the negatively charged drug forms a complex with the cationic lipid. The composition can increase the in vivo stability of the negatively charged drug after local or systemic administration and allows the intracellular delivery of the negatively charged drug. Thus, the composition will be useful for improving the therapeutic effect of the negatively charged drug.

Abstract: This invention relates to compounds, compositions, and methods useful for reducing HIF-1? target RNA and protein levels via use of dsRNAs, e.g., Dicer substrate siRNA (DsiRNA) agents.

Abstract: Libraries of nanoparticles comprising therapeutic agents and/or imaging agents are disclosed, as well as methods of making, customizing, and using such libraries of nanoparticles.

Abstract: A method for transfecting nucleic acid to cell comprising a step for forming a nucleic acid complex by bringing a double-stranded nucleic acid molecule into contact with a nucleic acid carrier having an amino acid sequence of alternating a basic amino acid and a hydrophobic amino acid, which has a peptide chain that forms a ?-sheet structure in which a side chain of a positively charged basic amino acid is disposed on one surface side and a side chain of a hydrophobic amino acid is disposed on the opposite surface side in the presence of the double-stranded nucleic acid molecule having a double helix structure, and by binding the double-stranded nucleic acid molecule and the peptide chain through either one or both of the electrostatic interaction between the side chains of the basic amino acid and phosphate groups and hydrogen bonds between the double stranded-nucleic acid molecule and the peptide chain and a nucleic acid complex used for the same are disclosed.

Abstract: The invention relates to lipid formulated double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a gene from the Ebola virus.

Abstract: The instant invention provides for novel cationic lipids that can be used in combination with other lipid components such as cholesterol and PEG-lipids to form lipid nanoparticles with oligonucleotides. It is an object of the instant invention to provide a cationic lipid scaffold that demonstrates enhanced efficacy along with lower liver toxicity as a result of lower lipid levels in the liver. The present invention employs low molecular weight cationic lipids with one short lipid chain to enhance the efficiency and tolerability of in vivo delivery of siRNA.

Abstract: Disclosed are lipophilic polynucleotide conjugates including polynucleotide-cholesterol conjugates and methods of delivering therapeutic polynucleotides to a mammalian cell or patient in need of treatment using said conjugates. The disclosure further provides methods of synthesizing the lipophilic polynucleotide conjugates. The conjugates are designed to mimic or target cellular miRNAs. The lipophilic moiety, such as cholesterol or cholesterol derivative, is spaced from the polynucleotide by a substantially linear hydrocarbon group. Due to an absence of significantly polar groups and/or exchangeable protons in the vicinity of the lipophilic moiety, the interaction between the lipophilic moiety and cell membranes is enhanced to provide for efficient entry into cells.

Abstract: An epidermal growth factor receptor (EGFR)-homing vector comprising a double-stranded RNA (dsRNA) molecule with an EGFR-binding peptide or polypeptide, is disclosed for use in combination with immune cells for treatment of cancer overexpressing EGFR.

Abstract: The present invention provides compositions comprising interfering RNA (e.g., siRNA, aiRNA, miRNA) that target polo-like kinase 1 (PLK-1) expression and methods of using such compositions to silence PLK-1 expression. More particularly, the present invention provides unmodified and chemically modified interfering RNA molecules which silence PLK-1 expression and methods of use thereof. The present invention also provides serum-stable nucleic acid-lipid particles (e.g., SNALP) comprising an interfering RNA molecule described herein, a cationic lipid, and a non-cationic lipid, which can further comprise a conjugated lipid that inhibits aggregation of particles. The present invention further provides methods of silencing PLK-1 gene expression by administering an interfering RNA molecule described herein to a mammalian subject. The present invention additionally provides methods of identifying and/or modifying PLK-1 interfering RNA having immunostimulatory properties.

Abstract: Systems and methods are described for parallel macromolecular delivery and biochemical/electrochemical interface to whole cells employing carbon nanostructures including nanofibers and nanotubes. A method includes providing a first material on at least a first portion of a first surface of a first tip of a first elongated carbon nanostructure; providing a second material on at least a second portion of a second surface of a second tip of a second elongated carbon nanostructure, the second elongated carbon nanostructure coupled to, and substantially parallel to, the first elongated carbon nanostructure; and penetrating a boundary of a biological sample with at least one member selected from the group consisting of the first tip and the second tip.

Abstract: This disclosure provides for compositions and methods for the use of designed nucleic acid-targeting nucleic acids, Argonautes, and complexes thereof.

Abstract: The present application relates to compositions comprising and methods of using a liposome comprising a pHLIP polypeptide, wherein a lipid bilayer of the liposome is substantially free of the pHLIP polypeptide.

Abstract: Provided herein are biodegradable copolymers and nanoplex delivery systems comprising the same and a cargo molecule, such as a nucleic acid, a polynucleotide or other biomolecule. The biodegradable copolymers may comprise a reducible polymer linearly modified with lysine, such as a linear lysine-modified N,N?-cystamine bisacrylamide. The biodegradable copolymers also may be conjugated to a sequestering moiety, such as dietheylamine. The biodegradable copolymers also may comprise one or both of a targeting moiety and one or more moieties to facilitate endosomal escape. Also provided are methods for treating a pathophysiological condition and for increasing biocompatibility of a polymeric delivery system upon delivery to a subject using the biodegradable copolymers and nanoplexes.

Abstract: The present invention is directed to adoptive immunotherapy using a lymphocyte in which an antigen-specific receptor and a bioactive material gene such as an IL-2 gene or a water-soluble TGF-beta receptor gene are transferred. The bioactive material is intensively secreted to, for example, a local site of a tumor, thereby reducing systemic side effects as much as possible, and the survival time of the lymphocyte is increased, thereby further improving the effect of the adoptive immunotherapy.

Abstract: The present invention is directed generally to eukaryotic host cells comprising artificial endosymbionts and methods of introducing artificial endosymbionts into eukaryotic host cells. The invention provides artificial endosymbionts that introduce a phenotype to host cells that is maintained in daughter cells. The invention additionally provides eukaryotic host cells containing magnetotactic bacteria.

Abstract: The present invention relates to a composition for improving the migration potential of a stem cell, a method for evaluating the migration potential of a stem cell and a method for screening an adjuvant of cell therapy improving the migration potential of a stem cell. The present invention may be effectively used for enhancing the efficacy of neurological disease-treatment by inducing therapeutic stem cells to migrate efficiently to the lesion site.

Abstract: Provided are a culture medium of an adipose-derived stem cell, a method for preparing the same, and a composition for promoting hair growth, in which the composition includes the culture medium. The adipose-derived stem cell (ADSC-T) according to the present invention exhibits long lifespan, improved cell proliferation rate, and extended proliferation period, as compared with a primary adipose-derived stem cell (ADSC), and thus, the adipose-derived stem cell (ADSC-T) can be usefully used for the study about the adipose-derived stem cell and the mass production of the culture medium of the adipose-derived stem cell. In addition, according to the present invention, the culture medium of the adipose-derived stem cell (ADSC-T) that expresses a T antigen of SV40 exhibits excellent hair growth effectiveness and can be usefully used as a raw material for the hair loss prevention and hair growing agents.

Abstract: The present invention relates to a new class of cationic polymers that self-assemble with a pH-sensitive dissolution switch, and their uses to deliver molecules of interest to a cell. The present invention also relates to compositions comprising said cationic polymers non-covalently associated with a molecule of interest, in particular with a siRNA.

Abstract: The present invention provides compositions comprising polytheylyene-dialkyloxypropyl conjugates (PEG-DAA), liposomes, SNALP, and SPLP comprising such compositions, and methods of using such compositions, liposomes, SNALP, and SPLP.

Abstract: The invention provides a nucleic-acid-transfecting composition which exhibits low cytotoxicity, which facilitates an effective nucleic acid transfection into a cell, and which improves expression of the nucleic acid in the cell. The composition for transfecting a nucleic acid into a cell, contains a di(C12-16 alkyl)dimethylammonium halide and a phospholipid.

Abstract: In certain embodiments, the invention provides methods for treating cancer, comprising: (a) obtaining a specimen of cancer tissue and normal tissue from a patient; (b) extracting total protein and RNA from the cancer tissue and normal tissue; (c) obtaining a protein expression profile of the cancer tissue and normal tissue; (d) identifying over-expressed proteins in the cancer tissue; (e) comparing the protein expression profile to a gene expression profile; (f) identifying at least one prioritized protein target by assessing connectivity of each said over-expressed protein to other cancer-related or stimulatory proteins; (g) designing a first RNA interference expression cassette to modulate the expression of at least one gene encoding the prioritized target protein; (h): designing a first RNA interference expression cassette to modulate the expression of at least one gene encoding a protein of higher priority in the signaling pathway in which the first protein is a component; (i) incorporating the first cass

Abstract: The embodiments herein provide a nanoparticle system for targeted gene delivery or a drug delivery and a method of synthesising the same. The nanoparticle composition for targeted gene transfer and drug delivery comprises a protein, a chitosan and a lipid. A method of synthesizing the nanoparticles involves preparing a gelatine and chitosan gel. A milky colloid solution is prepared with the gelatine, chitosan solution and a phosphatidylcholine. The milky colloid is homogenized for the self assembly of the nanoparticles. The milky colloid is subjected to high speed and high pressure homogenizer. The CHO cells are transfected with nanoparticles and lipofectamine 2000 for comparing the transfection efficiency. The nanoparticles deliver DNA, RNA, ribozyme and nucleotide sequences. The nanoparticles deliver lipophylic and hydrophilic drugs. The transfection efficiency of gene and drug is higher when the target cells are transferred with nanoparticles, compared to the cells transferred with lipofectamine 2000.

Abstract: The invention relates to enantiomerically pure DOTAP chloride and stable crystal modifications of (2R,S)-, (25)- and (2R)-DOTAP chloride, to a process for the preparation of these modifications, and to the use thereof as constituent for the preparation of medicaments.

Abstract: Compositions and methods for delivering cargo to cells are provided. One aspect provides a synthetic vesicle containing caveolin 1 or a fragment thereof in an amount effective to form lipid rafts in the vesicle. The synthetic vesicles can be used to deliver polynucleotides, proteins, therapeutic agents, or a combination thereof to specific membrane-bound compartments of a cell. In certain aspects, the synthetic vesicles can deliver cargo to cellular organelles such as mitochondria.

Abstract: The invention relates to a double-stranded ribonucleic acid (dsRNA) targeting a G-alpha q subunit (GNAQ) of a heterotrimeric G gene, and methods of using the dsRNA to inhibit expression of GNAQ.

Abstract: The present invention relates to a composition for enhancing an immune response, an epitope having immunogenicity, screening and preparing method thereof, a antibody to peptide antigen and screening and preparing method thereof. The composition of the present invention may be effectively used for preventing or treating diverse immune-deficiency diseases such as cancer, influenza virus, hepatitis C virus and RSV (respiratory syncytial virus) by enhancing immune responses.