Erythroid cells comprising phenylalanine ammonia lyase
AssigneeRubius Therapeutics, Inc.
InventorsAvak Kahvejian, Jordi Mata-Fink, John Round, David Arthur Berry, Noubar B. Afeyan
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Abstract
Compositions comprising synthetic membrane-receiver complexes, methods of generating synthetic membrane-receiver complexes, and methods of treating or preventing diseases, disorders or conditions therewith.
Claims27
An enucleated erythroid cell comprising an exogenous polypeptide comprising phenylalanine ammonia lyase (PAL) or a functional fragment thereof,
- wherein the enucleated erythroid cell is made by a process comprising introducing an exogenous nucleic acid encoding the exogenous polypeptide into a nucleated erythroid cell precursor.
A nucleated erythroid cell precursor comprising an exogenous polypeptide comprising PAL or a functional fragment thereof,
- wherein the nucleated erythroid cell precursor was made by a process comprising introducing an exogenous nucleic acid encoding the exogenous polypeptide into the nucleated erythroid cell precursor.
A method of reducing phenylalanine concentration in the blood of a subject, comprising administering to the subject a pharmaceutical composition comprising a plurality of enucleated erythroid cells comprising an exogenous polypeptide comprising PAL, wherein each of the enucleated erythroid cells of said plurality was made by a process comprising introducing an exogenous nucleic acid encoding the exogenous polypeptide into a nucleated erythroid cell precursor.
A method of reducing phenylalanine concentration in the blood of a subject, comprising administering to the subject a pharmaceutical composition comprising a plurality of nucleated erythroid cell precursors comprising an exogenous polypeptide comprising PAL, wherein each of the nucleated erythroid cell precursors of said plurality was made by a process comprising introducing an exogenous nucleic acid encoding the exogenous polypeptide into the nucleated erythroid cell precursor.
Dependencies are inferred from the claim text.