Abstract: The present invention relates to Adeno-associated virus type 9 methods and materials useful for intrathecal delivery of polynucleotides. Use of the methods and materials is indicated, for example, for treatment of lower motor neuron diseases such as SMA and ALS as well as Pompe disease and lysosomal storage disorders. It is disclosed that administration of a non-ionic, low-os-molar contrast agent, together with a rAA9 vector for the expression of Survival Motor Neuron protein, improves the survival of SMN mutant mice as compared to the administration of the expression vector alone.

Abstract: The present invention relates to the use of a milk whey protein hydrolysate for preventing or treating transepidermal water loss (TEWL) and/or TEWL-associated disorders or/and enhancing skin barrier function in young and adult mammals. The protein hydrolysate may be used in the prevention or treatment of TEWL-associated disorders, such as atopic dermatitis, dry or reactive skin or skin dehydration. It may also be used in the cosmetic use of improving skin appearance.

Abstract: Provided herein are peptide amphiphiles (PAs). In some embodiments, provided herein are PEGylated PAs (e.g. nonionic PAs). In some embodiments, the peptide amphiphiles are assembled into nanofibers. The nanofibers may further comprise a growth factor protein, which may bind to a growth factor binding sequence presented on the nonionic PA. In some embodiments, the nanofibers further comprise a charged peptide amphiphile. The charged peptide amphiphile may be bound to a growth factor protein. Further provided herein are methods of use of the peptide amphiphiles and compositions comprising the same, such as for regenerative therapy.

Abstract: Peptides and methods of use thereof, are disclosed for use in treating various disease and disorders, including inflammation, pain, oral mucositis, oral lesions, and cancer. The Peptides modulate the activity of the transcription factor NF ?B.

Abstract: The present invention relates to Adeno-associated virus type 9 methods and materials useful for intrathecal delivery of polynucleotides. Use of the methods and materials is indicated, for example, for treatment of lower motor neuron diseases such as SMA and ALS as well as Pompe disease and lysosomal storage disorders. It is disclosed that administration of a non-ionic, low-os-molar contrast agent, together with a rAA9 vector for the expression of Survival Motor Neuron protein, improves the survival of SMN mutant mice as compared to the administration of the expression vector alone.

Abstract: The present invention provides a treatment drug or prophylactic drug for diseases, disorders, or conditions related to endoplasmic reticulum (ER) stress. Specifically, the present invention provides a treatment drug or prophylactic drug for diseases, disorders, or conditions related to endoplasmic reticulum (ER) stress in the corneal epithelium, the drug containing a TGF?-signal inhibitor. As a preferred TGF?-signal inhibitor, the drug contains 4-[4-(1,3-benzodioxole-5-yl)-5-(2-pyridinyl)-1H-imidazole-2-yl]benzamide.

Abstract: Provided are various embodiments relating to compositions and methods for treating vascular disease, including core NOX1 promoters and variants thereof for regulating expression of transgenes in response to vascular pathology and allowing for increased transgene loading capacity. Also provided are variant FOXP polypeptides having a zinc finger and leucine zipper region of a different FOXP polypeptide. Further provided are vectors comprising the core NOX1 promoters and/or a coding sequence for variant FOXP polypeptides described herein and optionally coding sequence(s) for one or more additional therapeutic polypeptide(s), such as IL10, for treating inflammation-associated diseases, such as vascular disease. Also provided is a screening model for testing therapeutic agents capable of treating established and ongoing atherosclerotic pathology.

Abstract: Biomarkers tests which can be used to predict a positive or negative risk of preeclampsia are described. More specifically, a panel of biomarkers including MMP-7 and gpIIbIIIa, described. The test is useful to predict preeclampsia when a biological sample is obtained between the 16th and 22nd week of pregnancy. Prediction later in pregnancy can be achieved by a combination of Siglec-6, Activin A, ALCAM, and/or FCN2.

Abstract: A recombinant protein expressing one or more human growth factors, tumor antigens, and/or receptors or epitopes thereof on or within an immunogenic expression creating a recombinant protein in which one or more epitopes are presented on the surface of the sequence in their natural configuration. The growth factor, tumor antigen, and/or receptor, sequence(s) may be expressed within the encoding sequence at appropriate internal positions or at the termini as single expressions or as two or more tandem repeats.

Abstract: Peptides and methods of use thereof, are disclosed for use in treating various diseases and disorders, including inflammation, pain, oral mucositis, oral lesions, and cancer. The peptides modulate the activity of the transcription factor NF ?B.

Abstract: The present invention provides an agent, or a composition containing an agent, for use in treating or preventing a bacterial infection in a subject, wherein said agent comprises: (i) an oligopeptidic compound comprising a PCNA interacting motif and a domain that facilitates the cellular uptake of said compound, wherein the PCNA interacting motif is X1X2X3X4X5X6 (SEQ ID NO: 1) and wherein: X1 is a basic amino acid; X2 is an aromatic amino acid; X3 is an aromatic amino acid or a hydrophobic amino acid that has an R group comprising at least three carbon atoms; X4 is an uncharged amino acid other than an aromatic amino acid, Glycine (G) and Proline (P); X5 is any amino acid other than an acidic amino acid or an aromatic amino acid; and X6 is any amino acid other than an acidic amino acid or an aromatic amino acid, preferably a basic amino acid or Proline (P), wherein when X3 is not an aromatic amino acid, X5 is not lysine (K) and X6 is a basic amino acid or Proline (P); or (ii) a nucleic acid molecule comprising

Abstract: Provided are compositions and methods for potentiating the effect of antibiotics. The compositions comprise a non-covalent complex of alpha-lactalbumin and fatty acid. The fatty acids are cis, unsaturated, C14 to C20 fatty acids. The complex and antibiotic can be administered to an individual together or separately. The antibiotic may be one to which resistance has developed.

Abstract: This present application describes a therapeutic agent for treating acute or chronic graft-versus-host disease using clonal marrow stem cells (cMSCs) as active ingredient.

Abstract: The present disclosure generally relates to methods and compositions for treating skin. In some embodiments, the method uses an exfoliation tool. In some embodiments, the method uses an exfoliation tool together with selected skin care agents or compositions.

Abstract: The present invention concerns a pharmaceutical composition comprising an alpha-2 adrenergic receptor agonist for preventing or treating inflammatory pain and diseases in mucosa of oral cavity, pharynx and larynx. In another aspect the present invention provides mucosal bioadhesive slow release carriers for the extended and controlled release of alpha-2 adrenergic receptor agonists that can be used on mucosal surfaces for preventing or treating inflammatory pain and diseases in mucosa of oral cavity.

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: This invention relates, e.g., to a synthetic compound, Oxy149, having the structure (Formula I) or a bioactive or pharmaceutical composition comprising Oxy149 and a pharmaceutically acceptable carrier. Methods are also disclosed for using the compound or bioactive or pharmaceutical composition to treat a variety of disorders, including e.g. bone disorders, obesity, cardiovascular disorders, and neurological disorders. Oxy149 can be delivered either locally or systemically.

Abstract: Embodiments of the present invention are related to a method for producing a composition containing biologically active follistatin.

Abstract: The present invention relates to methods for the diagnosis and the treatment of familial thoracic aortic aneurysms caused by TGFB2 loss of function mutations. More particularly, the present invention relates to a method for determining whether a subject is predisposed to thoracic aortic aneurysms comprising detecting a TGFB2 loss of function mutation wherein the presence of the mutation indicated that the subject is predisposed to thoracic aortic aneurysms. The present invention also relates to a transforming growth factor beta-2 (TGF-?2) polypeptide for use in the prophylactic treatment of a subject who has been considered as predisposed to thoracic aortic aneurysms by the method of the invention (i.e. a subject having one TGB2 loss of function mutation according to the invention).

Abstract: The present invention relates to a method and kit for the classification and prognosis of wounds of mammalian, in particular in human. The method defines a molecular signature that enables one to characterize a pathological wound healing such as chronic or non-healing wound.

Abstract: Methods of coating bone surfaces and coated bones comprising a coating comprising (a) a porous polysaccharide scaffold and/or a plurality of polysaccharide nanofibers; and (b) a polyelectrolyte multilayer composition, are disclosed. The methods of coating bone surfaces and the coated bones disclosed provide a biomimetic periosteum to compensate for periosteum lost in preparing bone grafts, and the coated bone grafts, including allografts, restore lost osteogenic and osteoinductive qualities and improve clinical outcomes.

Abstract: The present invention relates to a device having osteoinductive and osteoconductive properties in vivo comprising a carrier containing calcium phosphate and an osteoinductive protein, wherein the carrier is homogenously coated with the protein. Moreover, the present invention relates to a method for the production of a device having osteoinductive and osteoconductive properties in vivo. The invention encompasses a pharmaceutical composition comprising the device of the invention or a device which is obtainable by the method of the invention and relates to the use of the device for the preparation of a pharmaceutical composition to be used for bone augmentation, for treating bone defects, for treating degenerative and traumatic disc disease, for treating bone dehiscence or to be used for sinus floor elevation. Finally, the invention relates to a kit comprising the device of the invention or a device which is obtainable by the method of the invention.

Abstract: A method for preparing a bioactive composition containing conditioned cell culture medium is disclosed. The method comprises culturing cells of two or more eukaryotic cell line to form conditioned culture media, separating the cultured cells from the conditioned culture media, and combining conditioned culture media to form a bioactive composition. Novel bioactive compositions, formulations and their use in treating of a variety of diseases and health conditions are also disclosed.

Abstract: A cyclized peptide designated BMP Binding Peptide (BBP) is a synthetic peptide that avidly binds rhBMP-2, as do endogenous forms of BBP, and sequence conservation between species results in a variety of useful BBP compositions. BBP increases the over-all osteogenic activity of rhBMP-2, increases the rate at which rhBMP-2 induces bone formation, and BBP induces calcification alone. Compositions and substrates including BBP, and methods of using BBP are useful in therapeutic, diagnostic and clinical applications requiring calcification and osteogenesis.

Abstract: A “nanolipogel” is a delivery vehicle including one or more lipid layer surrounding a hydrogel core, which may include an absorbent such as a cyclodextrin or ion-exchange resin. Nanolipogels can be constructed so as to incorporate a variety of different chemical entities that can subsequently be released in a controlled fashion. These different incorporated chemical entities can differ dramatically with respect to size and composition. Nanolipogels have been constructed to contain co-encapsulated proteins as well as small hydrophobic drugs within the interior of the lipid bilayer. Agents incorporated within nanolipogels can be released into the milieu in a controlled fashion, for example, nanolipogels provide a means of achieving simultaneous sustained release of agents that differ widely in chemical composition and molecular weight. Additionally, nanolipogels can favorably modulate biodistribution.

Abstract: The present invention relates to a GFP-CAP peptide having an amino acid sequence derived from TGF-?1 (transforming growth factor-?1) and a cell adhesion sequence, wherein the amino acid sequence derived from TGF-?1 consists of the amino acid sequence of SEQ ID NO:1 and the TGFP-CAP peptide is represented by the following formula I: Ile-Trp-Ser-Leu-Asp-Thr-Gln-Tyr-Cell adhesion sequence (I). The TGFP-CAP peptide of the present invention exhibits excellent anti-angiogenic activity. In addition, the TGFP-CAP peptide of the present invention prevents effectively melanin generation in skin to have skin whitening effects. The present peptide shows much higher stability and permeability to skin than natural-occurring TGF-?1. Such plausible activities and safety of the present peptide enable advantageously to application to drugs, quasi-drugs and cosmetics.

Abstract: Disclosed herein are an in situ-forming, bioadhesive hydrogel and the medical uses thereof. Being formed by in situ crosslinking through an enzymatic reaction, the hydrogel has an advantage over conventional bioadhesive hydrogels in terms of biocompatibility. In addition, the in situ-forming bioadhesive hydrogel has excellent biocompatibility and mechanical strength and has excellent tissue adhesiveness thanks to modification with/without dopa derivatives. The hydrogel finds a variety of applications in the biomedical field, including bioadhesives or hemostats, implant substances for tissue regeneration and augmentation, carriers for delivering biologically active materials or drugs, etc.

Abstract: The invention relates to novel biosynthetic growth factor mutants which exhibit improved biological activity.

Abstract: The present invention relates to methods of and compositions comprising cytokines for, improving the success rate of embryo implantation and the success rate of pregnancy rates in females, by providing an immunopermissive uterine environment prior to insemination or implantation of embryos. The methods of the present invention are used to make the uterus more receptive or less hostile to, for example, transferred embryos, sperm or other allografted tissue.

Abstract: The present invention is directed to a method of making a stable composition comprising growth factors, which may include platelet derived growth factors and transforming growth factors. The method comprises providing human umbilical cord blood plasma containing platelets, but substantially free of whole blood cells, and lysing the platelets to extrude growth factors into the plasma. The plasma may be obtained from multiple donors and pooled to form a homologous plasma mixture. In another embodiment, the growth factors are encapsulated in a liposome formed by a lipid bilayer, wherein the resulting composition remains stable and viable for at least 30 months. The present invention is also directed to the composition produced by the methods of the present invention and the process of applying the composition to a skin defect or wound.

Abstract: The present invention relates to a composition including stem cell-derived microvesicles as an active ingredient for promoting neurogenesis. The stem cell-derived microvesicles according to the present invention can promote neurogenesis and migration of nerves and also promote angiogenesis in vascular endothelial cells, and thus can be usefully used in treatment of neurological damage.

Abstract: Certain embodiments are directed to novel heterotrimeric fusions in which the ectodomain of the TGF-? type II receptor (T?P?II) is coupled to the N- and C-terminal ends of the endoglin-domain of the TGF-? type III receptor (TpRIIIE). Certain embodiments are directed to novel heterotrimeric polypeptides in which the ectodomain of the TGF-? type II receptor (TI3RII) is coupled to the N- and C-terminal ends of the endoglin-domain (E domain) of the TGF-? type III receptor (TI3RIII). This trimeric receptor, known as RER, can bind all three TGF-? isoforms with sub-nanomolar affinity and is effective at neutralizing signaling induced by all three TGF-? isoforms, but not other ligands of the TGF-? superfamily, such as activins, growth and differentiation factors (GDFs), and bone morphonogenetic proteins (BMPs).

Abstract: Described herein are compositions comprising a derivatized polyglucosamine and a small molecule, peptide, or protein and related methods of use, e.g., to deliver a small molecule, peptide, or protein to cells (e.g., cancer cells) or tissues (e.g., mucosal membrane and epithelial membrane), e.g., to treat a disease or condition in a subject.

Abstract: Methods of treating individuals with a glucose metabolism disorder and/or a body weight disorder, and compositions associated therewith, are provided.

Abstract: This invention provides a method of promoting bone healing by locally administering a vanadium-based insulin mimetic agent to a patient in need thereof. The invention also provides a new use of insulin-mimetic vanadium compounds for manufacture of medicaments for accelerating bone-healing processes. In addition, the invention also encompasses a bone injury treatment kit suitable for localized administration of insulin-mimetic vanadium compounds or compositions thereof to a patient in need of such treatment.

Abstract: The invention concerns a method for obtaining a highly enriched TGF-beta protein fraction in activated form, from a liquid solution rich in proteins said to be soluble in the aqueous phase of milk and/or of whey, said method comprising the following steps; a) adjusting soluble proteins purified at a concentration between 5 and 30 g/liter of solution; b) precipitating part of the whey proteins by acidic treatment of the solution thus obtained to a pH ranging between 4 and 5.5 and at a temperature ranging between 55° C. and 68° C.; c) carrying out a microfiltration of the treated solution by diafiltration, so as to obtain respectively a microfiltration retentate and a microfiltrate; d) recuperating the microfiltration retentate containing the protein fraction highly enriched in TGF-beta; e) drying the microfiltration retentate which has been subjected to diafiltration to obtain a powder highly enriched in TGF-beta.

Abstract: As the percentage of senior citizens increases at the present time, the purpose of the present invention is to provide a cosmetic which can maintain the skin in a healthy state by preventing damage to the skin that accompanies aging, more specifically, discoloration and wrinkling, is very safe, does not pose ethical problems, and can be supplied in a sufficient amount. The present invention provides a cosmetic that comprises as the main component a powder of the supernatant produced by culturing the bone marrow or dental pulp stem cells of a nonhuman mammal. Moreover, provided is a method for ion introduction for protein by which the cosmetic is introduced by ion introduction.

Abstract: Methods and compositions are disclosed for an intra-articular injection for the treatment of osteoarthritis. The methods and compositions comprising combinations of hyaluronic acid and a bone morphogenetic protein, like rhGDF-5, can be useful for any synovial joint, including the knee, shoulder, hip, ankle, hands, spinal facet, or temporomandibular joint, both for the relief of pain and for slowing disease progression.

Abstract: A radiation protection device for providing protection of a body part that includes active bone marrow from ionizing radiation may include a radiation protection component configured to be placed adjacent to and externally cover the body part so as to reduce a radiation dose absorbed in that body part.

Abstract: Disclosed herein are compositions and methods for treating or preventing a cardiac arrhythmia in a subject.

Abstract: The claimed invention provides a fusion polypeptide comprising a fibrous protein domain and a mineralization domain. The fusion is used to form an organic-inorganic composite. These organic-inorganic composites can be constructed from the nano- to the macro-scale depending on the size of the fibrous protein fusion domain used. In one embodiment, the composites can also be loaded with other compounds (e.g., dyes, drugs, enzymes) depending on the goal for the materials, to further enhance function. This can be achieved during assembly of the material or during the mineralization step in materials formation.

Abstract: The present invention is directed to GDF-5 related proteins having an improved capability of inducing cartilage formation and a reduced capability of inducing bone formation. The novel proteins are particularly useful in the treatment of cartilage defects, wherein the formation of bone tissue is undesirable.

Abstract: The present invention provides compositions comprising isolated human collagen, isolated human elastin and a pharmaceutically acceptable carrier wherein the human elastin is substantially insoluble in water with a molecular weight greater than 100 kDa. The present invention further provides methods and kits for soft tissue augmentation.

Abstract: The present invention is directed to stabilizing Bone Morphogenetic Protein in various lyophilized formulations and compositions. The present invention comprises formulations primarily including trehalose as an excipient for lyophilized compositions and their subsequent storage and reconstitution, and can also optionally include other excipients, including buffers and surfactants.

Abstract: Compositions of the invention for regenerating defective or absent myocardium comprise an emulsified or injectable extracellular matrix composition. The composition can also include an extracellular matrix scaffold component of any formulation, and further include added cells, proteins, or other components to optimize the regenerative process and restore cardiac function.

Abstract: The present invention provides compounds for disrupting the binding of a matrix metalloprotease (MMP) protein to a substrate protein at an interaction site other than the protease catalytic site. In particular the inventive compounds inhibit the MMP's ability to cleave a substrate protein. In some cases the compound may prevent activation of transforming growth factor beta (TGF?). The compounds are preferably polypeptide fragments of the hemopexin-like domain of the MMP, but may be mimetics thereof or peptides or mimetics of the portion of the MMP substrate protein to which the MMP interacts.

Abstract: Described herein is a cell tissue gel containing collagen and hyaluronan at a weight ratio of 0.01-100:1.

Abstract: The invention provides products of manufacture, e.g., biomaterials and implants, for cartilage maintenance and/or formation in-vivo, in-vitro, and ex-vivo, using nanotechnology, e.g., using nanotube, nanowire, nanopillar and/or nanodepots configured on surface structures of the products of manufacture.

Abstract: The present invention is directed to methods and compositions for accomplishing systemic delivery of minimally-soluble bioactive agents such as, but not limited to, proteins of the TGF-? superfamily via a peripheral mode of administration. According to the invention, an exemplary bioactive agent is BMP-7. The invention further provides for minimally-invasive systemic treatment of skeletal disorders such as osteoporosis as well as minimally-invasive systemic treatment of injured or diseased non-mineralized tissues and organs such kidneys. Practice of the invention eliminates adverse side effects at the peripheral site of intravenous administration of the bioactive agent.

Abstract: The present disclosure relates to compounds, compositions and methods for the treatment of diseases through inhibiting the activity of the transforming growth factor beta (TGF-?). More specifically, the disclosed compounds, compositions and methods are useful in the treatment of certain cancers (e.g. multiple myeloma, hematologic malignancies), diseases associated with excessive TGF-? activity including fibrosis, dermal scarring, immune dysfunction, and bone loss by inhibiting the conversion of latent TGF-? to active TGF-?. A method of preventing the activation of TGF-? in pathology is also provided, comprising administering an amount of the compounds sufficient to inhibit conversion of latent TGF-? to active TGF-? by thrombospondinl (TSP1), resulting in reduced TGF-? activity and reduced adverse effects such as fibrosis, bone loss, and immune dysfunction.