Compositions and methods for introducing double-stranded breaks within the SERPINA1 gene are provided. Compositions and methods for reducing and eliminating mutant forms of α1-antitrypsin (AAT), such as seen in subjects having α1-antitrypsin deficiency (AATD), are provided.

Compositions that increase the bioavailability of dihydromyricetin are presented. The bioavailability is increased by methods including formulating dihydromyricetin in nanoparticle form, delivering dihydromyricetin with permeabilizers, and encapsulating dihydromyricetin with an enteric coating.

A novel composite, and research on the preparation, application and the like of the composite. The method for preparing the composite comprises: contacting a polyinosinic-polycytidylic acid, at least one cationic stabilizer, and a soluble calcium salt in a liquid reaction system, the cationic stabilizer being a water-soluble non-antibiotic amino compound having a molecular weight of less than or equal to 5 kDa, or a graft copolymer formed by a water-soluble non-antibiotic amino compound and one or more of methoxypolyethylene glycol, polyethylene glycol, polyethylenimine, folic acid, or galactose. The composite has moderate viscosity and molecular weight, is convenient to use in pharmaceutical application, has stable chemical properties, is not easy to be degraded in long-term storage, and is safe to use. The composite, if used alone, can significantly enhance the non-specific immune response of the body and achieve the purpose of preventing and treating diseases, and other drugs, and can achieve better anti-tumor, anti-viral and anti-(super) bacteria efficacy and is easily absorbed by patients, if used in combination with other drugs.

The present disclosure offers therapeutic solutions to cancer patients up to now considered as unable to undergo a standard-of-care treatment involving radiotherapy or at high risk to undergo a standard-of-care treatment involving radiotherapy. The disclosure relates to nanoparticles and/or aggregates of nanoparticles for use in the treatment of cancer in such a patient, wherein the nanoparticles and/or aggregates of nanoparticles preferably comprise more than 30% by weight of at least one chemical element having an atomic number (Z) between 20 and 83. The disclosed treatments involve a step of administering the nanoparticles and/or aggregates of nanoparticles to the patient, and a step of exposing the patient to a total dose of ionizing radiations that is equal to or less than 85% of the total dose delivered in the standard-of-care treatment. The present description also discloses new compositions comprising such nanoparticles and/or aggregates of nanoparticles as well as uses thereof.

The presently disclosed subject matter provides compositions, methods, and kits for transfecting adipose-derived mesenchymal stem cells (AMSCs) in freshly extracted adipose tissue using nanoparticles comprising biodegradable polymers self-assembled with nucleic acid molecules. The presently disclosed subject matter also provides methods for treating a neurological disease in a patient in need thereof, the method comprising administering the AMSCs transfected with the nucleic acid molecules to the patient, wherein the nucleic acid molecules encode one or more bioactive molecules functional in the treatment of a neurological disease, particularly wherein the neurological disease is a brain tumor.

Compositions and methods for introducing double-stranded breaks within the SERPINA1 gene are provided. Compositions and methods for reducing and eliminating mutant forms of α1-antitrypsin (AAT), such as seen in subjects having α1-antitrypsin deficiency (AATD), are provided.

Present disclosure relates to a solvent-free method of encapsulating a hydrophobic active in personal care, hydrophobic active in crop protection or a hydrophobic active pharmaceutical ingredient (API) in polymeric nanoparticles. The method includes mixing the hydrophobic active in a polymer melt, wherein the polymer melt comprises a melt of a block co-polymer, wherein the polymer melt acts as a solvent for the hydrophobic active. The method further includes maintaining the polymer melt in water for sufficient time to allow self-assembly of the block co-polymer to encapsulate the hydrophobic active therein.

Compositions of nanolipid carrier molecules comprising PEG molecules and solid and liquid lipids wherein the PEG has a molecular weight of between about 1000 and 5000 are described. Methods of administering nanolipid carrier molecules are also described. Also described is a method for treating fungal ocular infections by topical ocular administration of nanolipid carrier molecules comprising PEG molecules and solid and liquid lipids wherein the PEG has a molecular weight of between about 1000 and 5000.

The presently disclosed subject matter provides a kinetically controlled mixing process, referred to herein as “flash nanocomplexation” or “(FNC),” to accelerate the mixing of a polyanion solution, for example, a plasmid DNA solution, with a polycation solution to match the polyelectrolyte complex (PEC) assembly kinetics through turbulent mixing in a microchamber, thus achieving explicit control of the kinetic conditions for nanoparticle assembly as demonstrated by the tunability of nanoparticle size, composition, hydrodynamic size, hydrodynamic density, surface charge, and polyanion payload.

The invention relates to a novel use of ultrafine nanoparticles, of use as a diagnostic, therapeutic or theranostic agent, characterized by their mode of administration via the airways. The invention is also directed toward the applications which follow from this novel mode of administration, in particular for imaging the lungs, and the diagnosis or prognosis of pathological pulmonary conditions. In the therapeutic field, the applications envisioned are those of radiosensitizing or radioactive agents for radiotherapy (and optionally curietherapy), or for neutron therapy, or of agents for PDT (photodynamic therapy), in particular for the treatment of lung tumors.