Patent classifications
C07K14/14
POLYNUCLEOTIDES ENCODING GALACTOSE-1-PHOSPHATE URIDYLYLTRANSFERASE FOR THE TREATMENT OF GALACTOSEMIA TYPE 1
The invention relates to mRNA therapy for the treatment of galactosemia type 1 (Gal-1). mRNAs for use in the invention, when administered in vivo, encode human galactose-1-phosphate uridylyltransferase (GALT), isoforms thereof, functional fragments thereof, and fusion proteins comprising GALT. 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 GALT expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient GALT activity in subjects, namely galactose-1-phosphate (Gal-1-P).
METHOD FOR PRODUCING ARTIFICIAL RECOMBINANT ROTAVIRUS
Provided is a method for producing an artificial recombinant virus of the family Reoviridae, the method comprising the steps of: (1) introducing a FAST protein expression vector and/or a capping enzyme expression vector into host cells; (2) introducing a vector containing expression cassettes for individual RNA genome segments of a virus or introducing a set of single-stranded RNA transcripts from the expression cassettes into host cells; and (3) culturing the host cells.
The method of the present invention allows more efficient production of an artificial recombinant virus of the family Reoviridae as compared with conventional methods and allows artificial recombinant rotavirus production without using a helper virus.
METHOD FOR PRODUCING ARTIFICIAL RECOMBINANT ROTAVIRUS
Provided is a method for producing an artificial recombinant virus of the family Reoviridae, the method comprising the steps of: (1) introducing a FAST protein expression vector and/or a capping enzyme expression vector into host cells; (2) introducing a vector containing expression cassettes for individual RNA genome segments of a virus or introducing a set of single-stranded RNA transcripts from the expression cassettes into host cells; and (3) culturing the host cells.
The method of the present invention allows more efficient production of an artificial recombinant virus of the family Reoviridae as compared with conventional methods and allows artificial recombinant rotavirus production without using a helper virus.
Rotavirus particles with chimeric surface proteins
The present invention relates to the use of rotavirus particles for displaying a heterologous protein, alone or in complex with another molecule. The invention further relates to methods that employ these modified rotavirus particles to rapidly determine the structure of the heterologous protein or the complex using cryo-electron microscopy (cryo-EM). The invention also relates to a method of immunizing a patient, wherein said method comprises administering to the patient the modified rotavirus particles of the invention.
Rotavirus particles with chimeric surface proteins
The present invention relates to the use of rotavirus particles for displaying a heterologous protein, alone or in complex with another molecule. The invention further relates to methods that employ these modified rotavirus particles to rapidly determine the structure of the heterologous protein or the complex using cryo-electron microscopy (cryo-EM). The invention also relates to a method of immunizing a patient, wherein said method comprises administering to the patient the modified rotavirus particles of the invention.
Method for producing artificial recombinant rotavirus
Provided is a method for producing an artificial recombinant virus of the family Reoviridae, the method comprising the steps of: (1) introducing a FAST protein expression vector and/or a capping enzyme expression vector into host cells; (2) introducing a vector containing expression cassettes for individual RNA genome segments of a virus or introducing a set of single-stranded RNA transcripts from the expression cassettes into host cells; and (3) culturing the host cells. The method of the present invention allows more efficient production of an artificial recombinant virus of the family Reoviridae as compared with conventional methods and allows artificial recombinant rotavirus production without using a helper virus.
Method for producing artificial recombinant rotavirus
Provided is a method for producing an artificial recombinant virus of the family Reoviridae, the method comprising the steps of: (1) introducing a FAST protein expression vector and/or a capping enzyme expression vector into host cells; (2) introducing a vector containing expression cassettes for individual RNA genome segments of a virus or introducing a set of single-stranded RNA transcripts from the expression cassettes into host cells; and (3) culturing the host cells. The method of the present invention allows more efficient production of an artificial recombinant virus of the family Reoviridae as compared with conventional methods and allows artificial recombinant rotavirus production without using a helper virus.
Avian reovirus vaccines
The present invention relates to novel strains of avian reovirus that were isolated from clinical cases of viral arthritis/tenosynovitis in chickens in the southeast United States. The invention is directed to these novel group 1 and group 2 avian reoviruses, diagnostic assays using antibodies and/or nucleotide- or amino acid-specific components of such viruses, such as the S1 gene encoding the sigma C protein, and to vaccines that protect chickens from disease caused by such viruses.
Polynucleotides encoding galactose-1-phosphate uridylyltransferase for the treatment of galactosemia type 1
The invention relates to mRNA therapy for the treatment of galactosemia type 1 (Gal-1). mRNAs for use in the invention, when administered in vivo, encode human galactose-1-phosphate uridylyltransferase (GALT), isoforms thereof, functional fragments thereof, and fusion proteins comprising GALT. 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 GALT expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient GALT activity in subjects, namely galactose-1-phosphate (Gal-1-P).
Polynucleotides encoding galactose-1-phosphate uridylyltransferase for the treatment of galactosemia type 1
The invention relates to mRNA therapy for the treatment of galactosemia type 1 (Gal-1). mRNAs for use in the invention, when administered in vivo, encode human galactose-1-phosphate uridylyltransferase (GALT), isoforms thereof, functional fragments thereof, and fusion proteins comprising GALT. 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 GALT expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient GALT activity in subjects, namely galactose-1-phosphate (Gal-1-P).