Provided herein is a dual vector transfection system for the production of recombinant adeno-associated virus (rAAV). The dual vector transfection system generally comprises: (1) a first nucleic acid vector comprising a first nucleotide sequence encoding an AAV Rep protein, a second nucleotide sequence comprising an rAAV genome comprising a transgene, and a third nucleotide sequence encoding an AAV capsid protein; and (2) a second nucleic acid vector comprising a helper virus gene.

Provided herein is a dual vector transfection system for the production of recombinant adeno-associated virus (rAAV). The dual vector transfection system generally comprises: (1) a first nucleic acid vector comprising a first nucleotide sequence encoding an AAV Rep protein, a second nucleotide sequence comprising an rAAV genome comprising a transgene, and a third nucleotide sequence encoding an AAV capsid protein; and (2) a second nucleic acid vector comprising a helper virus gene.

This disclosure relates to mRNA therapy for the treatment of glycogen storage disease type 1a, (GSD-Ia), and related symptoms such as hypoglycemia. mRNAs for use in the invention, when administered in vivo, encode human glucose-6-phosphatase (G6Pase or G6PC), and functional fragments and variants thereof. 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 G6PC expression and/or activity in subjects. mRNA therapies of the invention further increase the glucose production, and reduce the abnormal accumulation of glycogen and/or glucose-6-phosphate associated with GSD-Ia.

This disclosure relates to mRNA therapy for the treatment of glycogen storage disease type 1a, (GSD-Ia), and related symptoms such as hypoglycemia. mRNAs for use in the invention, when administered in vivo, encode human glucose-6-phosphatase (G6Pase or G6PC), and functional fragments and variants thereof. 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 G6PC expression and/or activity in subjects. mRNA therapies of the invention further increase the glucose production, and reduce the abnormal accumulation of glycogen and/or glucose-6-phosphate associated with GSD-Ia.

Provided herein, inter alia, are engineered phytase polypeptides and fragments thereof with improved thermotolerance as well as methods for producing and using the same for enhancing animal performance on one or more metrics.

Provided herein, inter alia, are engineered phytase polypeptides and fragments thereof with improved thermotolerance as well as methods for producing and using the same for enhancing animal performance on one or more metrics.

The present invention provides, among other things, methods and compositions for production of recombinant I2S protein with improved potency and activity using cells co-express I2S and FGE protein. In some embodiments, cells according to the present invention are engineered to simultaneously over-express recombinant I2S and FGE proteins. Cells according to the invention are adaptable to various cell culture conditions. In some embodiments, cells of the present invention adaptable to a large-scale suspension serum-free culture.

The present invention provides, among other things, methods and compositions for production of recombinant I2S protein with improved potency and activity using cells co-express I2S and FGE protein. In some embodiments, cells according to the present invention are engineered to simultaneously over-express recombinant I2S and FGE proteins. Cells according to the invention are adaptable to various cell culture conditions. In some embodiments, cells of the present invention adaptable to a large-scale suspension serum-free culture.

The present invention generally relates to the microbiological industry, and specifically to the production of L-serine or L-serine derivatives using genetically modified bacteria. The present invention provides genetically modified microorganisms, such as bacteria, wherein the expression of genes encoding for enzymes involved in the degradation of L-serine is attenuated, such as by inactivation, which makes them particularly suitable for the production of L-serine at higher yield. The present invention also provides means by which the microorganism, and more particularly a bacterium, can be made tolerant towards higher concentrations of serine. The present invention also provides methods for the production of L-serine or L-serine derivative using such genetically modified microorganisms.

This invention relates to the treatment of cancer in an individual by administration of a population of modified T cells that express a recombinant cAMP phosphodiesterase (PDE) or a fragment thereof and an antigen receptor which binds specifically to cancer cells in the individual. Populations of modified T cells and methods of producing populations of modified T cells are provided, along with pharmaceutical compositions and methods of treatment