In some aspects, the disclosure relates to GDF/BMP antagonists and methods of using GDF/BMP antagonists to treat, prevent, or reduce the progression rate and/or severity of pulmonary hypertension (PH), particularly treating, preventing or reducing the progression rate and/or severity of one or more PH-associated complications. The disclosure also provides methods of using a GDF/BMP antagonist to treat, prevent, or reduce the progression rate and/or severity of a variety of conditions including, but not limited to, pulmonary vascular remodeling, pulmonary fibrosis, and right ventricular hypertrophy. The disclosure further provides methods of using a GDF/BMP antagonist to reduce right ventricular systolic pressure in a subject in need thereof.

In some aspects, the disclosure relates to GDF/BMP antagonists and methods of using GDF/BMP antagonists to treat, prevent, or reduce the progression rate and/or severity of pulmonary hypertension (PH), particularly treating, preventing or reducing the progression rate and/or severity of one or more PH-associated complications. The disclosure also provides methods of using a GDF/BMP antagonist to treat, prevent, or reduce the progression rate and/or severity of a variety of conditions including, but not limited to, pulmonary vascular remodeling, pulmonary fibrosis, and right ventricular hypertrophy. The disclosure further provides methods of using a GDF/BMP antagonist to reduce right ventricular systolic pressure in a subject in need thereof.

Polypeptides that are glutathione-S-transferases originating from different schistosome parasites, as well as nucleic acids, vectors, compositions or kits, for use in the preventive or therapeutic treatment of vasculitis or of a disease characterized by a M1/M2 macrophage ratio dysregulation, such as e.g. a decrease of the M1-type immune response and/or an increase of the M2-type immune response.

Polypeptides that are glutathione-S-transferases originating from different schistosome parasites, as well as nucleic acids, vectors, compositions or kits, for use in the preventive or therapeutic treatment of vasculitis or of a disease characterized by a M1/M2 macrophage ratio dysregulation, such as e.g. a decrease of the M1-type immune response and/or an increase of the M2-type immune response.

A nanocomplex, 50 to 1000 nm in size, containing a lipid-like nanoparticle formed of a cationic lipid-based compound and a modified protein formed of a protein and an anionic polymer that includes a plurality of polar groups, the lipid-like nanoparticle and the modified protein being non-covalently bonded to each other. Also disclosed are a method of preparing the above-described nanocomplex and use thereof for treating a medical condition. Further disclosed is a pharmaceutical composition containing a nanocomplex.

A nanocomplex, 50 to 1000 nm in size, containing a lipid-like nanoparticle formed of a cationic lipid-based compound and a modified protein formed of a protein and an anionic polymer that includes a plurality of polar groups, the lipid-like nanoparticle and the modified protein being non-covalently bonded to each other. Also disclosed are a method of preparing the above-described nanocomplex and use thereof for treating a medical condition. Further disclosed is a pharmaceutical composition containing a nanocomplex.

Methods and compositions for modulating repeat non-ATG protein (RAN protein) translation are provided. In some aspects, the disclosure provides methods of inhibiting RAN protein translation by contacting a cell with an effective amount of an inhibitor of eIF2 phosphorylation or an inhibitor of protein kinase R (PKR). In some embodiments, methods described by the disclosure are useful for treating diseases associated with RAN protein translation, such as certain neurodegenerative diseases.

Methods and compositions for modulating repeat non-ATG protein (RAN protein) translation are provided. In some aspects, the disclosure provides methods of inhibiting RAN protein translation by contacting a cell with an effective amount of an inhibitor of eIF2 phosphorylation or an inhibitor of protein kinase R (PKR). In some embodiments, methods described by the disclosure are useful for treating diseases associated with RAN protein translation, such as certain neurodegenerative diseases.

The present invention provides stable, dry powder messenger RNA formulations for therapeutic use, and methods of making and using the same.

The present invention provides stable, dry powder messenger RNA formulations for therapeutic use, and methods of making and using the same.