The disclosure relates, in some aspects, to compositions and methods for treatment of diseases associated with aberrant lysosomal function, for example Parkinson's disease (PD) and Gaucher disease. In some embodiments, the disclosure provides expression constructs comprising a transgene encoding beta-Glucocerebrosidase (GBA) or a portion thereof alone or in combination with one or more PD-associated genes. In some embodiments, the disclosure provides methods of Parkinson's disease by administering such expression constructs to a subject in need thereof.

The present invention relates to three-dimensional self-assembled nucleic acid nanoparticles, a drug delivery system comprising the same, and a pharmaceutical composition for the prevention or treatment of acute kidney injury, which comprises the same. The three-dimensional self-assembled nucleic acid nanoparticles of the present invention, which have a tetrahedral structure, exhibit an excellent renal-targeting ability, and thus the nanoparticles conjugated with the pharmaceutically active ingredient for p53 exhibit excellent p53 and caspase 3 expression reductions in vitro and in vivo, and can thereby be applied to the prevention or treatment of acute kidney injury.

The disclosure relates, in some aspects, to compositions and methods for treatment of diseases associated with aberrant lysosomal function, for example Parkinson's disease (PD) and Gaucher disease. In some embodiments, the disclosure provides expression constructs comprising a transgene encoding beta-Glucocerebrosidase (GBA) or a portion thereof alone or in combination with one or more PD-associated genes. In some embodiments, the disclosure provides methods of Parkinson's disease by administering such expression constructs to a subject in need thereof.

The disclosure relates, in some aspects, to compositions and methods for treatment of diseases associated with aberrant lysosomal function, for example Parkinson's disease (PD) and Gaucher disease. In some embodiments, the disclosure provides expression constructs comprising a transgene encoding beta-Glucocerebrosidase (GBA) or a portion thereof alone or in combination with one or more PD-associated genes. In some embodiments, the disclosure provides methods of Parkinson's disease by administering such expression constructs to a subject in need thereof.

A protein selected from the group consisting of Chrdl1, Fam3c, Fam3b and a fragment thereof, or a polynucleotide encoding therefor, for use in treating or reducing the risk of heart disease.

This document relates to methods and materials involved in treating a mammal having a gastrointestinal disorder (e.g., an irritable bowel syndrome (IBS) such as post-infection IBS (PI-IBS)). For example, one or more protease inhibitors and/or one or more microorganisms that produce one or more protease inhibitors can be administered to a mammal having, or at risk of developing, a gastrointestinal disorder to treat the mammal.

The present invention relates to methods of treating chemotherapy-induced peripheral neuropathy. In particular, the methods provide a new way of reducing neuropathic pain associated with chemotherapy-induced peripheral neuropathy by administering a nucleic acid construct encoding human HGF proteins. This application further provides nucleic acid constructs, pharmacological compositions, and methods of administration of the nucleic acid constructs that are effective in treating the neuropathic pain.

The present invention provides a method for treating a fibrotic lung disease or fibrotic lung condition in a subject that involves an increase in NEU1 expression and/or activity, comprising administering to the subject an effective amount of an agent that inhibits the activity of NEU 1 sialidase, thereby treating the fibrotic lung disease or fibrotic lung condition in the subject.

Provided are genetically modified non-human animals that express a human or chimeric (e.g., humanized) IL33, and methods of use thereof.

Apicomplexan parasites or red blood cells infected with apicomplexan parasites are administered to an animal in combination with a delayed death agent that initially allows parasite replication but subsequently kills the apicomplexan parasites. This allows the elicitation of an immune response by the animal while preventing the parasites producing a serious infection of the animal. The apicomplexan parasites may be malaria or babesia parasites. The delayed death agent may be a tetracycline class antibiotic, a macrolide antibiotic or a lincosamide antibiotic.