Abstract: The invention relates to dose escalation enzyme replacement therapy using acid sphingomyelinase (ASM) for the treatment of human subjects having acid sphingomyelinase deficiency (ASMD), and, in particular, patients with non-neurological manifestations of Niemann-Pick Disease (NPD), and in certain embodiments, NPD type B.

Abstract: The present invention is a composition and methods of administering the composition to improve a person's mental and physical health by increasing energy levels, motivation, willingness to communicate, appetite, and memory. The composition may be used to treat mental conditions by improving a person's wiliness and ability to communicate. The increase in appetite effect of the composition may be used to treat eating disorders. The composition comprises a carbohydrate, one or more NAD+/NADP+ producing vitamins, poly-sulfurous chelating agents, vasodilating salts, coenzyme-A producing vitamins, antioxidants, kinins, and biological energy sources.

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 present invention provides a pharmaceutical composition formulated for oral delivery, comprising a particulate non-covalently associated mixture of pharmacologically inert silica nanoparticles having a hydrophobic surface, a polysaccharide, and a biologically active protein or peptide suspended in an oil. The present invention further provides methods of manufacturing same and therapeutic methods utilizing same for oral delivery of a therapeutic protein or peptide.

Abstract: Described herein are compositions which include digestive enzymes and which are formulated to reduce one or more symptoms of a neuropsychiatric disorder. Also described herein is a method for treating an individual with a neuropsychiatric disorder using digestive enzymes and their derivatives to alleviate the symptoms of neuropsychiatric disorders. The method comprises administering to the individual an effective amount of digestive enzymes that are either naturally or recombinantly derived, or their derivatives, in an amount effective to reduce one or more symptoms of the neuropsychiatric disorder.

Abstract: A process is provided for releasably encapsulating a biological entity. The process comprises combining a solution of a surfactant in a non-polar solvent with a precursor material and the biological entity to form an emulsion. The emulsion comprises a polar phase dispersed in a non-polar phase, wherein the polar phase comprises the biological entity. The particles comprising the biological entity are then formed from the polar phase.

Abstract: Provided is a method for monitoring a gene mutation associated with a cancer in a patient over time. Also provided is a method of selecting and/or applying treatment or therapy for a subject.

Abstract: Compounds of general formula (I): R1-Wn-Xm-AA1-AA2-AA3-AA4-AA5-AA6-AA7-AA8-AA9-Yp-Zq-R2 (I) their stereoisomers, mixtures thereof and/or their cosmetically or pharmaceutically acceptable salts, preparation processes, cosmetic or pharmaceutical compositions which contain them and their use in medicine, particularly in the treatment and/or prevention of pain, inflammation, itching, neurological, compulsive and/or neuropsychiatric diseases and/or disorders and in processes of treatment and/or care of the skin, hair and/or mucous membranes mediated by neuronal exocytosis.

Abstract: A method for enhancing the growth, increasing feed utilization and reducing mortality in poultry (such as chickens and turkeys) by adding a pomegranate-derived additive to their feed. Pomegranate-derived additive and its use for enhancing growth of poultry without growth-promoting antibiotics.

Abstract: This invention relates to phytases, polynucleotides encoding them, uses of the polynucleotides and polypeptides of the invention, as well as the production and isolation of such polynucleotides and polypeptides. In particular, the invention provides polypeptides having phytase activity under high temperature conditions, and phytases that retain activity after exposure to high temperatures. The phytases of the invention can be thermotolerant and/or thermostable at low temperatures, in addition to higher temperatures. The phytases of the invention can be used in foodstuffs to improve the feeding value of phytate rich ingredients. The phytases of the invention can be formulated as foods or feeds or supplements for either to, e.g., aid in the digestion of phytate. The foods or feeds of the invention can be in the form of pellets, liquids, powders and the like.

Abstract: The present invention relates generally to methods of treating cancer with arginine deiminase, and in particular pegylated arginine deiminase.

Abstract: A protein composition which comprises a mixture of glycine, phenylalanine and histidine and/or a cellulose ether derivative as an additive and has resistance to radiation sterilization.

Abstract: The pharmaceutical use of lipases related to the Thermomyces lanuginosus (Humicola lanuginosa) lipase comprising amino acids 1-269 of SEQ ID NO: 2, optionally in combination with a protease and/or an amylase. Examples of medical indications are: Treatment of digestive disorders, pancreatic exocrine insufficiency (PEI), pancreatitis, cystic fibrosis, diabetes type I, and/or diabetes type II. The lipases of the invention have, e.g., an improved digestion performance in vitro, an improved activity at a pH in the neutral range, an improved stability at low pH, and are stable against protease-degradation, and/or are stable in the presence of pepsin and bile salts. The invention also relates to methods of determining digestion performance in vitro of lipases, as well as to certain novel variants of the lipase of T. lanuginosus.

Abstract: A sterile composition which comprises a protein and an aliphatic polyester containing the protein and is sterilized with radiation. In this sterile composition, the structure and function (activity) of the protein are retained.

Abstract: The present invention relates to a method for treating or preventing pathogenic infections in a subject having Cystic Fibrosis, COPD, and/or an open wound. This method involves selecting a subject having Cystic Fibrosis, COPD, and/or an open wound and administering to the selected subject a ceramidase under conditions effective to reduce ceramide and to treat or prevent the pathogenic infection. The method also involves the use of a ceramidase in combination with other drugs to reduce infection, reduce ceramide, or improve lung function in Cystic Fibrosis, COPD, and/or open wound patients.

Abstract: Provided is a preventive, progression inhibitor or remedy for a disease one of the causes of which is the activation of the P13K/AKT signaling pathway or vice versa. A phosphorylation-inhibiting and/or dephosphorylating agent, which has an effect of inhibiting the phosphorylation at least at one of the phosphorylation sites of PTEN protein selected from the group consisting of T382, T383 and S380 and/or an effect of dephosphorylating the same, is prepared. Alternatively, a phosphorylation-inhibiting or dephosphorylating agent for PTEN is screened by a method comprising a step for confirming an ability of a test substance to inhibit the phosphorylation at least at one of the phosphorylation sites of PTEN protein selected from the group consisting of T382, T383 and S380 or a dephosphorylation ability thereof. Then, a substance having an effect opposite to the inhibition of PTEN phosphorylation or dephosphorylation thereof, e.g.

Abstract: A therapeutic agent for the treatment of toxemia, preeclampsia and eclampsia and a method for preparing the therapeutic agent are disclosed. The therapeutic agent is a stable pharmaceutical preparation containing, but not limited to, digestive/pancreatic enzymes. The therapeutic agent may be manufactured by a variety of encapsulation technologies. Delivery of the therapeutic agent may be made orally, through injection, by adherence of a medicated patch or by other methods. Further, a method of using the presence of chymotrypsin in the maternal GI tract as a biomarker, to determine the likelihood of developing preeclampsia, a pregnancy induced hypertension, and eclampsia/toxemia is disclosed.

Abstract: The present invention is directed to compositions and methods of preserving viability of islets of Langerhans for transplantation, and treating various disease and other abnormal or pathological conditions, including inflammatory bowel disease, ischemic heart disease, acute lung injury, acute respiratory distress syndrome and radiation-induced brain injury, with DNA repair enzymes that are directed to the mitochondria.

Abstract: Compositions containing conjugates of heparosan polymer with at least one drug are disclosed, along with methods of production and use thereof.

Abstract: Described are methods of inhibiting cytohesin-2 to treat cancer, treat diabetic nephropathy, and deliver a compound into a cell.

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: A therapeutic agent for the treatment of the symptoms of addiction and the method for preparing the therapeutic agent is disclosed. The therapeutic agent is a stable pharmaceutical preparation containing, but not limited to, digestive/pancreatic enzymes. The therapeutic agent may be manufactured by a variety of encapsulation technologies. Delivery of the therapeutic agent may be made orally, through injection, by adherence of a medicated patch or other method. Further, a method of using of a biomarker, the presence of chymotrypsin in the gastrointestinal tract to determine the presence of symptoms of addiction, and the likelihood of relapsing into addiction is disclosed.

Abstract: A method for treating cancer using an intra-patient dose escalation procedure to deliver dosages of Crotalus durissus terrificus venom.

Abstract: Methods of treating, reducing, or preventing lung infections or lung inflammation include identifying a patient in need of treatment and administering a therapeutically effective amount of at least one cationic steroid antimicrobial (CSA), or a pharmaceutically acceptable salt thereof. Treatment of cystic fibrosis lung infections, COPD lung infections, inflammation of the lungs in these patient populations, and lung scarring in these patient populations is also described.

Abstract: The present technology relates to cosmetic and pharmaceutical compositions useful for minimizing the appearance of aging via a safe method of stimulating sirtuin activity. In particular, the present technology relates to compositions and methods that utilize a combination of a sirtuin activator with a sirtuin-offsetting agent in order to maintain a healthy population of skin cells while preventing survival and growth of abnormal cells that are irreparably damaged.

Abstract: The present invention provides a method for retarding unhealthy manifestations brought by ageing of human beings (in particular, but not limited to the reduction of sexual activity and fertility, climax, changes in glucose tolerance, reduction of cognitive and mnestic functions, reduction of stress resistance, development of organ and tissue sclerosis). According to the invention this task is solved by administration into the blood circulation of an agent which inactivates extracellular blood plasma DNA e.g. a DNase enzyme.

Abstract: Methods and therapeutic treatments of diseases such as viral infections are provided including applying peg-Arginase I. Methods are provided that treat inflammation mediated diseases with peg-Arginase I.

Abstract: The invention provides a method for treating cancer in a subject or a method of inducing an anti-tumour effect including reducing tumour volume, inhibiting or slowing tumour growth, inhibiting tumour progression, altering the metabolic activity of a tumour, inducing quiescence of a tumour, inhibiting or reducing metastasis, inhibiting or reducing tumour invasiveness, reducing tumour weight, reducing tumour neovascularisation, improving time to disease progression (TDP) and/or improving survival, the method comprising administering to the subject an effective amount of angiogenin or an angiogenin agonist.

Abstract: The invention relates to deoxyribonuclease for use in the treatment of a suspected or existing C. difficile infection; a pharmaceutical or veterinary composition or formulation comprising at least deoxyribonuclease for use in the treatment of a suspected or existing C. difficile infection; a combination therapeutic comprising at least deoxyribonuclease for use in the treatment of a suspected or existing C. difficile infection; a method of treating a mammal suspected of being infected with, or infected with, C. difficile comprising the use of at least deoxyribonuclease; a method of cleaning or sterilising a material or product comprising the use of at least deoxyribonuclease; and a cleaning or sterilising product impregnated with or containing at least deoxyribonuclease.

Abstract: The present invention relates to certain amido-benzyl sulfoxide and sulfone compounds, pharmaceutical compositions comprising such compounds, and methods of treatment using such compounds.

Abstract: The present invention relates to the use of ribonucleases (RNases) in the treatment or prevention of disease.

Abstract: Compositions of modulators of acyl-CoA lysocardiolipin acyf transferase 1 (ALCAT1) expression, function or activity are provided. In particular, inhibitors of ALCAT1 are useful in treating metabolic diseases, cardiac diseases and, in general diseases associated with mitochondrial dysfunction. Assays for identification of novel ALCAT1 modulators are provided.

Abstract: Recombinant human lysosomal acid lipase (rhLAL) containing an N-terminal truncation, a composition of truncated recombinant human LAL (TLAL), an isolated mixture comprising TLAL and at least one other form of rhLAL are disclosed. A method of purifying TLAL from a mixture of LAL proteins, pharmaceutical compositions comprising TLAL and methods of producing TLAL are further disclosed.

Abstract: The invention provides for engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are vectors and vector systems, some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors with additional functional domains. Also provided are methods of directing CRISPR complex formation in prokaryotic and eukaryotic cells to ensure enhanced specificity for target recognition and avoidance of toxicity.

Abstract: A method of using vaults as carrier molecules to deliver one or more than one substance to an organism, or to a specific tissue or to specific cells, or to an environmental medium. A vault-like particle. A method of preventing damage by one or more than one substance to an organism, to a specific tissue, to specific cells, or to an environmental medium, by sequestering the one or more than one substance within a vault-like particle. A method of delivering one or more than one substance or a sensor to an organism, to a specific tissue, to specific cells, or to an environmental medium. According to another embodiment of the present invention, there is provided a method of making vault-like particles, and making vault-like particles comprising one or more than one substance, or one or more than one sensor.

Abstract: The invention provides for engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are vectors and vector systems, some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors with additional functional domains. Also provided are methods of directing CRISPR complex formation in prokaryotic and eukaryotic cells to ensure enhanced specificity for target recognition and avoidance of toxicity.

Abstract: Methods and compositions are provided for increasing responsiveness to therapeutic metalloproteases including increasing and/or maximizing responsiveness and preventing botulinum and tetanus toxin resistance due to a functional deficiency of zinc. Also provided are methods for zinc replacement or supplement in lacking individuals comprising the administration of a zinc supplement for a loading period and/or administration of a phytase supplement together with the zinc supplement. Also provided are methods for standardization of botulinum toxin potency assays that provide for greater certainty and margins of safety in the use of products from different manufacturers.

Abstract: The present invention provides, among other things, compositions, kits and methods for subcutaneous delivery of lysosomal enzymes for effective treatment of lysosomal storage diseases. In some embodiments, the present invention provides methods for treating Hunter syndrome by subcutaneous administration of a replacement iduronate-2-sulfatase (I2S) protein. In some embodiments, the present invention provides a kit comprising an arrangement of components for subcutaneously administering iduronate-2-sulfatase (I2S) protein.

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

Abstract: Provided is a pharmaceutical composition for treating corneal endothelium wounds, the composition including angiogenin as an effective ingredient. The present invention relates to a new topical therapeutic use of angiogenin for treatment of corneal endothelium wounds, wherein the angiogenin that is generally known to be involved in angiogenesis activates a PI3K/Akt/eNOS pathway thereof in ocular corneal endothelium to increase migration and proliferation of corneal endothelial cells that are not capable of self-proliferation and to promote prevention and treatment of corneal endothelial cell wounds.

Abstract: The present invention relates to a method to increase the absorption by a human infant of at least one unsaturated fatty acid, said method comprising the enteral administration to said infant of recombinant human bile-salt-stimulated lipase (rhBSSL). In another aspect the invention also relates to a method to improve the visual and/or cognitive development of a human infant, said method comprising the enteral administration to said infant of rhBSSL. Such methods have particular utility for preterm human infants, particular those in medical need of increasing their absorption of or availability to such unsaturated fatty acids. In further aspects, the invention relates to kits, packaged-pharmaceutical-products, recombinant human bile-salt-stimulated lipase and pharmaceutical compositions, in each case useful for increasing the absorption by a human infant of at least one unsaturated fatty acid, or for increasing the visual and/or cognitive development of a human infant.

Abstract: The present invention relates to a method to increase the growth velocity of a human infant, said method comprising the enteral administration to said infant of recombinant human bile-salt-stimulated lipase (rhBSSL). Such method has particular utility for underweight or preterm human infants, particular those in medical need of increasing their growth velocity. The invention also relates to compositions, including infant feeds, kits, packaged-pharmaceutical-products and pharmaceutical compositions, and also to methods to prepare infant feeds.

Abstract: A process for preparing a triglyceride composition comprising from 50 to 80% by weight StOSt and from 5 to 20% by weight StOO comprises reacting triolein with stearic acid in the presence of a 1,3-specific lipase from Rhizopus oryzae to form interesterified glycerides and fractionating the interesterified triglycerides.

Abstract: The invention discloses a kit which comprises a formulation containing artemisinin or the derivatives thereof, a formulation containing ribonuclease, and a specification.

Abstract: Disclosed herein are transcription activator-like effector nuclease (TALEN)-related compositions and methods of using said TALENs for correcting mutant genes.

Abstract: An aqueous composition having increased protein stability is obtained by: a. determining a pH at which the protein has stability at the desired temperature; b. adding to the composition at least one displacement buffer wherein the displacement buffer has a pKa that is at least 1 unit greater or less than the pH of step (a); and c. adjusting the pH of the composition to the pH of step (a); wherein the aqueous composition does not comprise a conventional buffer at a concentration greater than about 2 mM and wherein the conventional buffer has a pKa that is within 1 unit of the pH of step (a).

Abstract: Compositions, methods, strategies, kits, and systems for the supercharged protein-mediated delivery of functional effector proteins into cells in vivo, ex vivo, or in vitro are provided. Compositions, methods, strategies, kits, and systems for delivery of functional effector proteins using cationic lipids and cationic polymers are also provided. Functional effector proteins include, without limitation, transcriptional modulators (e.g., repressors or activators), recombinases, nucleases (e.g., RNA-programmable nucleases, such as Cas9 proteins; TALE nuclease, and zinc finger nucleases), deaminases, and other gene modifying/editing enzymes. Functional effector proteins include TALE effector proteins, e.g., TALE transcriptional activators and repressors, as well as TALE nucleases.

Abstract: A concentrated inhalable formulation of an antibiotic drug such as ciprofloxacin is disclosed. The antibiotic is formulated with sodium acetate and liposome which incorporate antibiotic. The formulation is aerosolized and inhaled for treatment of respiratory tract infections and other medical conditions.

Abstract: High affinity SIRP-? reagent are provided, which (i) comprise at least one amino acid change relative to the wild-type protein; and (ii) have an increased affinity for CD47 relative to the wild-type protein. Compositions and methods are provided for modulating phagocytosis in a mammal by administering a therapeutic dose of a pharmaceutical composition comprising a high affinity SIRP? reagent, which blocks the physiological binding interaction between SIRP? and its ligand CD47.

Abstract: Compositions, methods, strategies, kits, and systems for the supercharged protein-mediated delivery of functional effector proteins into cells in vivo, ex vivo, or in vitro are provided. Compositions, methods, strategies, kits, and systems for delivery of funcational effector proteins using cationic lipids and cationic polymers are also provided. Functional effector proteins include, without limitation, transcriptional modulators (e.g., repressors or activators), recombinases, nucleases (e.g., RNA-programmable nucleases, such as Cas9 proteins; TALE nuclease, and zinc finger nucleases), deaminases, and other gene modifying/editing enzymes. Functional effector proteins include TALE effector proteins, e.g., TALE transcriptional activators and repressors, as well as TALE nucleases.