RNA interference-based methods and products for inhibiting the expression of mutant Glycyl-tRNA Synthetase (GARS) genes are provided. Delivery vehicles such as recombinant adeno-associated viruses deliver DMAs encoding GARS microRNAs, as well as a replacement GARS gene that is resistant to knock down by the microRNAs. The methods have application in the treatment of N diseases or disorders associated with mutant GARS including, but not limited to, Charcot-Marie-Tooth Disease Type 2D (CMT2D).

Disclosed herein are methods and compositions for the treatment and/or prevention of diseases or conditions comprising administration of a therapeutic biological molecule, and/or naturally or artificially occurring derivatives, analogues, or pharmaceutically acceptable salts thereof, alone or in combination with one or more active agents (e.g., an aromatic-cationic peptide). The present technology provides compositions related to aromatic-cationic peptides linked to a therapeutic biological molecule and uses of the same. In some embodiments, the aromatic-cationic peptide comprises 2′,6′-dimethyl-Tyr-D-Arg-Phe-Lys-NH.sub.2, Phe-D-Arg-Phe-Lys-NH.sub.2, or D-Arg-2′,6′-Dmt-Lys-Phe-NH.sub.2.

Disclosed herein are methods and compositions for the treatment and/or prevention of diseases or conditions comprising administration of a therapeutic biological molecule, and/or naturally or artificially occurring derivatives, analogues, or pharmaceutically acceptable salts thereof, alone or in combination with one or more active agents (e.g., an aromatic-cationic peptide). The present technology provides compositions related to aromatic-cationic peptides linked to a therapeutic biological molecule and uses of the same. In some embodiments, the aromatic-cationic peptide comprises 2′,6′-dimethyl-Tyr-D-Arg-Phe-Lys-NH.sub.2, Phe-D-Arg-Phe-Lys-NH.sub.2, or D-Arg-2′,6′-Dmt-Lys-Phe-NH.sub.2.

The present invention relates to a composition for treatment or prevention of infectious inflammatory diseases comprising tryptophanyl-tRNA synthetase as an active ingredient, and a composition for immune enhancement. More specifically, the present invention relates to a pharmaceutical composition for treatment or prevention of infectious inflammatory diseases comprising tryptophanyl-tRNA synthetase as an active ingredient, a food composition for preventing or improving, a veterinary composition for preventing or treating, and a composition for immune enhancement comprising a tryptophanyl-tRNA synthetase as an active ingredient, respectively.

The composition of the present invention can be effectively used for preventing or treating diseases of humans and animals caused by infection from bacteria, viruses or fungi and the like by inhibiting infections such as bacterial, viral, and fungal infections at an early stage particularly through activating innate immune response.

The present invention relates to a composition for treatment or prevention of infectious inflammatory diseases comprising tryptophanyl-tRNA synthetase as an active ingredient, and a composition for immune enhancement. More specifically, the present invention relates to a pharmaceutical composition for treatment or prevention of infectious inflammatory diseases comprising tryptophanyl-tRNA synthetase as an active ingredient, a food composition for preventing or improving, a veterinary composition for preventing or treating, and a composition for immune enhancement comprising a tryptophanyl-tRNA synthetase as an active ingredient, respectively.

The composition of the present invention can be effectively used for preventing or treating diseases of humans and animals caused by infection from bacteria, viruses or fungi and the like by inhibiting infections such as bacterial, viral, and fungal infections at an early stage particularly through activating innate immune response.

Compositions capable of enhancing and/or eliciting an immune response in a subject and methods of using the compositions. The compositions are capable of enhancing an IgA immune response and/or an IgG immune response and comprise an agent capable of reducing the level of binding of ATP to a P2X7 receptor to a subject. The compositions are for oral administration.

Compositions capable of enhancing and/or eliciting an immune response in a subject and methods of using the compositions. The compositions are capable of enhancing an IgA immune response and/or an IgG immune response and comprise an agent capable of reducing the level of binding of ATP to a P2X7 receptor to a subject. The compositions are for oral administration.

The present invention provides an improved process for lipid nanoparticle formulation and mRNA encapsulation. In some embodiments, the present invention provides a process of encapsulating messenger RNA (mRNA) in lipid nanoparticles comprising a step of mixing a suspension of preformed lipid nanoparticles and mRNA.

The present invention relates to the use of glutamine synthetase as a protein therapy (such as enzyme replacement protein therapy) for the treatment of hyperammonemia. In particular the invention relates to the systemic administration of glutamine synthetase. The glutamine synthetase may be provided in conjugated or fusion form, to increase its half-life in the circulation. Also provided is a pharmaceutical composition comprising glutamine synthetase. The invention also relates to the uses, methods and compositions involving a combination of the glutamine synthetase protein and an ammonia lowering agent, such as a nitrogen scavenger.

The present invention relates to the use of glutamine synthetase as a protein therapy (such as enzyme replacement protein therapy) for the treatment of hyperammonemia. In particular the invention relates to the systemic administration of glutamine synthetase. The glutamine synthetase may be provided in conjugated or fusion form, to increase its half-life in the circulation. Also provided is a pharmaceutical composition comprising glutamine synthetase. The invention also relates to the uses, methods and compositions involving a combination of the glutamine synthetase protein and an ammonia lowering agent, such as a nitrogen scavenger.