Abstract: Described herein are lipid nanoparticles (LNPs), comprising neutral or positively charged nucleic acid mimics (NPNAMs) and optionally nucleic acids, and compositions thereof. Also described are methods of preparing LNPs comprising NPNAMs, and methods of use for intracellular gene editing. In particular, the LNPs comprising NPNAMs and optionally nucleic acids may be used in methods for the correction and/or treatment of a genetic disorder, disease, or condition in a subject.

Abstract: This invention pertains to the field of biopolymer-loaded (where the biopolymer(s) are active pharmaceutical ingredient(s) (API(s)) polymer-based nanoparticles formulated, for example, for curative, therapeutic prophylactic and/or diagnostic applications. The polymer used to formulate the nanoparticles can be any biodegradable synthetic polymer or combination of polymers, including combinations of PLGA and PEG. Biopolymers used as active pharmaceutical ingredients (API) can include natural and unnatural nucleic acids such as DNA, RNA and LNA. Biopolymers used as active pharmaceutical ingredients (APIs) can also include neutral and positively charged nucleic acid mimics (NPNAM) such as for example, peptide nucleic acids, morpholinos, pyrrolidine-amide oligonucleotide mimics, morpholinoglycine oligonucleotides and methyl phosphonates and derivatives thereof. In some embodiments, the nanoparticles are loaded with both nucleic acids and NPNAMs as the APIs.

Abstract: Disclosed are methods for treating bleeding disorders, such as hemophilia, in subjects in need thereof by administering an antibody that specifically binds CD73. The methods reduce production of adenosine, increase platelet activation and/or enhance the level of coagulation on the platelet surface to reduce and/or stop bleeding. In some embodiments, the methods can further include co-administering Factor VIII to treat the bleeding disorder.

Abstract: Disclosed are methods for treating bleeding disorders, such as hemophilia, in subjects in need thereof by administering an antibody that specifically binds CD73. The methods reduce production of adenosine, increase platelet activation and/or enhance the level of coagulation on the platelet surface to reduce and/or stop bleeding. In some embodiments, the methods can further include co-administering Factor VIII to treat the bleeding disorder.

Abstract: Methods of treating bleeding disorders, such as bleeding diseases such as hemophilia, by administering adenosine 2a receptor and/or adenosine 2b receptor antagonists to subjects in need thereof are disclosed. In some embodiments, the methods further include administration of the antagonist with one or more of Factor VIII, Factor IX and Factor XI to treat the bleeding disorder.

Abstract: Compositions for tissue engineering and drug delivery have been developed based on solutions of two or more polymers which form semi-interpenetrating or interpenetrating polymer networks upon exposure to active species following injection at a site in a patient in need thereof. The polymers crosslink to themselves but not to each other; semi-interpenetrating networks are formed when only one of the polymers crosslink. The resulting viscous solutions retain the biologically active molecules or cells at the site of injection until release or tissue formation, respectfully, occurs. As a result of studies conducted with polymer-cell suspensions forming interpenetrating polymer networks, it has been determined that polymer solutions can be formulated wherein the active species is provided by exposure of the polymer solution to an exogenous source of active species, typically electromagnetic radiation, preferably light.

Abstract: A method for temporarily adhering an image to a surface is shown utilizing liquid soluble substrates that have printed images of liquid insoluble ink. The liquid soluble substrate is positioned on a surface, such as a billboard, sidewalk, parking lot, telephone pole, skin, clothing, etc., and a liquid, typically water, is applied to the liquid soluble substrate and/or to the surface in such a quantity, and at such a temperature, and at such a pressure and velocity so as to be absorbed by the liquid soluble substrate in a sponge-like fashion. As the substrate dries, it takes on the shape of the surface, conforming and adhering to the surface through a mechanical interlock therewith. Various coatings may be applied to extend the longevity of the substrate. The liquid soluble substrate may be removed from the surface by applying a sufficient quantity of liquid at a sufficient temperature, pressure and velocity.

Abstract: Compositions for tissue engineering and drug delivery have been developed based on solutions of two or more polymers which form semi-interpenetrating or interpenetrating polymer networks upon exposure to active species following injection at a site in a patient in need thereof. The polymers crosslink to themselves but not to each other; semi-interpenetrating networks are formed when only one of the polymers crosslink. The resulting viscous solutions retain the biologically active molecules or cells at the site of injection until release or tissue formation, respectfully, occurs. As a result of studies conducted with polymer-cell suspensions forming interpenetrating polymer networks, it has been determined that polymer solutions can be formulated wherein the active species is provided by exposure of the polymer solution to an exogenous souce of active species, typically electromagnetic radiation, preferably light.