Provided are compositions comprising newly identified protein fragments of aminoacyl-tRNA synthetases, polynucleotides that encode them and complements thereof, related agents, and methods of use thereof in diagnostic, drug discovery, research, and therapeutic applications.

The present invention provides PEGylated aspartyl-tRNA synthetase (DRS) polypeptides, compositions comprising the same, and methods of using such polypeptides and compositions for treating or diagnosing a variety of conditions. The PEGylated DRS polypeptides of the invention have improved controlled release properties, stability, half-life, and other pharmacokinetic properties compared to non-PEGylated DRS polypeptides.

Angelman syndrome is a genetic neurological disorder with characteristics including delayed development, intellectual disability, severe speech impairment, and problems with movement and balance. Provided herein are polynucleotides, vectors, polypeptides, cells, compositions, kits and methods to treat Angelman syndrome.

Angelman syndrome is a genetic neurological disorder with characteristics including delayed development, intellectual disability, severe speech impairment, and problems with movement and balance. Provided herein are polynucleotides, vectors, polypeptides, cells, compositions, kits and methods to treat Angelman syndrome.

This disclosure relates to mRNA therapy for the treatment of propionic acidemia (PA). mRNAs for use in the invention, when administered in vivo, encode human propionyl-CoA carboxylase alpha (PCCA) and/or human propionyl-CoA carboxylase beta (PCCB), and isoforms thereof, functional fragments thereof, and fusion proteins comprising PCCA and/or PCCB. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of propionyl-CoA carboxylase (PCC) expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of disease-associated toxic metabolites associated with deficient PCCA or PCCB activity, in subjects.

This disclosure relates to mRNA therapy for the treatment of propionic acidemia (PA). mRNAs for use in the invention, when administered in vivo, encode human propionyl-CoA carboxylase alpha (PCCA) and/or human propionyl-CoA carboxylase beta (PCCB), and isoforms thereof, functional fragments thereof, and fusion proteins comprising PCCA and/or PCCB. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of propionyl-CoA carboxylase (PCC) expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of disease-associated toxic metabolites associated with deficient PCCA or PCCB activity, in subjects.

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 diseasess 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 diseasess 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.

Provided herein are compositions methods for the treatment and/or prevention of liver injury. In particular, carbamoyl phosphate synthetase-1 (CPS-I) peptides and polypeptides (e.g., enzymatically active or inactive CPS-I peptides and polypeptides), and methods of use thereof for the treatment and/or prevention of liver injury are provided.

The present invention provides the peptide nucleic acid derivative which targets 5′ splice site of the human ACC2 pre-mRNA “exon 12”. The peptide nucleic acid derivatives in the present invention strongly induce splice variants of the human ACC2 mRNA in cell and are very useful to treat conditions or disorders of skin aging associated with the human ACC2 protein.