The present invention relates to the treatment of fatty liver disease, by administration of glutamine synthetase.

The present invention relates to the treatment of fatty liver disease, by administration of glutamine synthetase.

The present invention relates to a composition comprising the UNE-L domain of leucyl-tRNA synthetase as an effective ingredient for increasing muscle. More particularly, the UNE-L domain of LRS according to the present invention, which is a region that controls activity of Vps34, activates mTORC1 involved in protein synthesis and increases myocyte differentiation and muscle fiber regeneration, thus finding useful application in muscle augmentation.

The present invention relates to a composition comprising the UNE-L domain of leucyl-tRNA synthetase as an effective ingredient for increasing muscle. More particularly, the UNE-L domain of LRS according to the present invention, which is a region that controls activity of Vps34, activates mTORC1 involved in protein synthesis and increases myocyte differentiation and muscle fiber regeneration, thus finding useful application in muscle augmentation.

Synthetic polynucleotides encoding human propionyl-CoA carboxylase alpha (synPCCA) and exhibiting augmented expression in cell culture and/or in a subject are described herein. Adeno-associated viral (AAV) gene therapy vectors encoding synPCCA successfully rescued the neonatal lethal phenotype displayed by propionyl-CoA carboxylase alpha (Pcca.sup.−/−) deficient mice, lowered circulating methylcitrate levels in the treated animals, and resulted in prolonged hepatic expression of the product of the synPCCA transgene in vivo.

Synthetic polynucleotides encoding human propionyl-CoA carboxylase alpha (synPCCA) and exhibiting augmented expression in cell culture and/or in a subject are described herein. Adeno-associated viral (AAV) gene therapy vectors encoding synPCCA successfully rescued the neonatal lethal phenotype displayed by propionyl-CoA carboxylase alpha (Pcca.sup.−/−) deficient mice, lowered circulating methylcitrate levels in the treated animals, and resulted in prolonged hepatic expression of the product of the synPCCA transgene in vivo.

The disclosure relates to compositions and methods for providing chemoprotective effects to target cells.

The disclosure relates to compositions and methods for providing chemoprotective effects to target cells.

The disclosure relates to compositions and methods for providing chemoprotective effects to target cells.

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).