Patent classifications
C07K14/805
METHODS TO TREAT MITOCHONDRIAL-ASSOCIATED DYSFUNCTIONS OR DISEASES
Methods for the treatment of disorders associated with mitochondrial dysfunction, including rare, inborn errors of metabolism caused by genetic mutations; neurodegenerative disease; diabetes; and aging and age-associated decline. Generally, the methods include administering a therapeutically effective amount of one or more treatments that (i) induce hemoglobin “left-shifting” and/or (ii) induce anemia, as described herein, to a subject who is in need of, or who has been determined to be in need of, such treatment.
METHODS OF PROTEIN PURIFICATION
Provided herein are methods for purifying proteins from mixtures of proteins using ultrafiltration, such as tangential-flow filtration (TFF). Provided herein are methods for isolating an apoprotein from a protein solution comprising a conjugated protein, wherein the conjugated protein comprises the apoprotein and a hydrophobic ligand associated with the apoprotein.
ANIMAL CELL LINES FOR FOODS CONTAINING CULTURED ANIMAL CELLS
Modified cell lines and methods for use in the production of cultured meat are disclosed. The inventive methods provide for insertion of cell cycle regulatory genes or genes that encode for animal myoglobin into the genome of an animal cell to proliferate and flavor cell productions, followed by excising the inserted genes to terminate proliferation.
ANIMAL CELL LINES FOR FOODS CONTAINING CULTURED ANIMAL CELLS
Modified cell lines and methods for use in the production of cultured meat are disclosed. The inventive methods provide for insertion of cell cycle regulatory genes or genes that encode for animal myoglobin into the genome of an animal cell to proliferate and flavor cell productions, followed by excising the inserted genes to terminate proliferation.
PHARMACEUTICAL COMPOSITIONS COMPRISING GENE-CORRECTED PRIMARY CELLS
In certain aspects, the present invention provides methods for inducing a stable gene modification of a target nucleic acid via homologous recombination in a primary cell, such as a primary blood cell and/or a primary mesenchymal cell. In certain other aspects, the present invention provides methods for enriching a population of genetically modified primary cells having targeted integration at a target nucleic acid. The methods of the present invention rely on the introduction of a DNA nuclease such as a Cas polypeptide and a homologous donor adeno-associated viral (AAV) vector into the primary cell to mediate targeted integration of the target nucleic acid. Also provided herein are methods for preventing or treating a disease in a subject in need thereof by administering to the subject any of the genetically modified primary cells or pharmaceutical compositions described herein to prevent the disease or ameliorate one or more symptoms of the disease.
PHARMACEUTICAL COMPOSITIONS COMPRISING GENE-CORRECTED PRIMARY CELLS
In certain aspects, the present invention provides methods for inducing a stable gene modification of a target nucleic acid via homologous recombination in a primary cell, such as a primary blood cell and/or a primary mesenchymal cell. In certain other aspects, the present invention provides methods for enriching a population of genetically modified primary cells having targeted integration at a target nucleic acid. The methods of the present invention rely on the introduction of a DNA nuclease such as a Cas polypeptide and a homologous donor adeno-associated viral (AAV) vector into the primary cell to mediate targeted integration of the target nucleic acid. Also provided herein are methods for preventing or treating a disease in a subject in need thereof by administering to the subject any of the genetically modified primary cells or pharmaceutical compositions described herein to prevent the disease or ameliorate one or more symptoms of the disease.
NUCLEASE-MEDIATED GENOME EDITING OF PRIMARY CELLS AND RELATED METHODS OF TREATMENT
In certain aspects, the present invention provides methods for inducing a stable gene modification of a target nucleic acid via homologous recombination in a primary cell, such as a primary blood cell and/or a primary mesenchymal cell. In certain other aspects, the present invention provides methods for enriching a population of genetically modified primary cells having targeted integration at a target nucleic acid. The methods of the present invention rely on the introduction of a DNA nuclease such as a Cas polypeptide and a homologous donor adeno-associated viral (AAV) vector into the primary cell to mediate targeted integration of the target nucleic acid. Also provided herein are methods for preventing or treating a disease in a subject in need thereof by administering to the subject any of the genetically modified primary cells or pharmaceutical compositions described herein to prevent the disease or ameliorate one or more symptoms of the disease.
SYSTEMS AND METHODS FOR MANUFACTURE OF ENDOTOXIN-FREE HEMOGLOBIN-BASED DRUG SUBSTANCE
The present disclosure relates to methods and systems for manufacturing stabilized hemoglobin solutions. The methods and systems incorporate single use components for endotoxin-free formulation. The hemoglobin solutions may be substantially endotoxin-free and/or highly deoxygenated.
SYSTEMS AND METHODS FOR MANUFACTURE OF ENDOTOXIN-FREE HEMOGLOBIN-BASED DRUG SUBSTANCE
The present disclosure relates to methods and systems for manufacturing stabilized hemoglobin solutions. The methods and systems incorporate single use components for endotoxin-free formulation. The hemoglobin solutions may be substantially endotoxin-free and/or highly deoxygenated.
Multi-functional chemical agents, and the method for protein modification
A multifunctional chemical agents comprising functional agents Fn1, Fn2 and linkers, for the linchpin directed (LDM), protein directed (PDPM) modifications of proteins, and Fn1 accelerated kinetic labeling by Fn2.