C12Y403/01024

Bacteria engineered to treat a disease or disorder

Genetically programmed microorganisms, such as bacteria, pharmaceutical compositions thereof, and methods of modulating and treating a disease and/or disorder are disclosed.

ENGINEERED PHENYLALANINE AMMONIA LYASE POLYPEPTIDES

The present invention provides engineered phenylalanine ammonia-lyase (PAL) polypeptides and compositions thereof, as well as polynucleotides encoding the engineered phenylalanine ammonia-lyase (PAL) polypeptides.

SYNTHETIC MEMBRANE-RECEIVER COMPLEXES

Compositions comprising synthetic membrane-receiver complexes, methods of generating synthetic membrane-receiver complexes, and methods of treating or preventing diseases, disorders or conditions therewith.

ENGINEERED PHENYLALANINE AMMONIA LYASE POLYPEPTIDES

The present invention provides engineered phenylalanine ammonia-lyase (PAL) polypeptides and compositions thereof, as well as polynucleotides encoding the engineered phenylalanine ammonia-lyase (PAL) polypeptides.

ENGINEERED PHENYLALANINE AMMONIA LYASE ENZYMES

Aspects of the disclosure relate to phenylalanine ammonia lyase (PAL) enzymes, including engineered PAL enzymes, and their use in catalyzing chemical reactions.

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Human PAH expression cassette for treatment of PKU by liver-directed gene replacement therapy
12173308 · 2024-12-24 · ·

Provided herein are expression cassettes for expressing a transgene in a liver cell, wherein the transgene encodes a PAH polypeptide. Also provided are methods to treat phenylketonuria (PKU) and/or to reduce levels of phenylalanine in an individual in need thereof. Further provided herein are vectors (e.g., rAAV vectors), viral particles, pharmaceutical compositions and kits for expressing a PAH polypeptide in an individual in need thereof.

COMPOSITIONS AND METHODS FOR TREATING PHENYLKETONURIA

Phenylketonuria (PKU) is caused by a mutation in the phenylalanine hydroxylase (P AH) gene in the liver, Provided herein are compositions and methods for treating phenylketonuria (PKU) and related disorders. Polynucleotides for expressing bacterial and plant-derived phenylalanine ammonia lyase (PAL) are provided herein. Also provided herein are methods of treating PKU and related disorders that include administration of polynucleotides encoding PAL proteins.

METHOD FOR THE BIOSYNTHESIS OF DIOSMIN AND/OR HESPERIDIN IN A MICROORGANISM

The present invention relates to a recombinant microorganism which is modified to be capable of producing diosmin and hesperidin and to the use thereof for producing diosmin and/or hesperidin.

SYNTHETIC MEMBRANE-RECEIVER COMPLEXES

Compositions comprising synthetic membrane-receiver complexes, methods of generating synthetic membrane-receiver complexes, and methods of treating or preventing diseases, disorders or conditions therewith.

BACTERIA ENGINEERED TO REDUCE HYPERPHENYLALANINEMIA

Genetically engineered bacteria, pharmaceutical compositions thereof, and methods of modulating and treating diseases associated with hyperphenylalaninemia are disclosed.