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US 9,644,180 B2Granted
Synthetic membrane-receiver complexes
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.
Claims21
A method of treating a subject having PKU comprising:
- a) obtaining an isolated population of functional erythroid cells comprising a receiver polypeptide, wherein said receiver polypeptide comprises phenylalanine ammonia lyase (PAL), wherein the functional erythroid cells of said isolated population comprising said receiver polypeptide are not hypotonically loaded cells, and
- b) administering to a subject in need thereof a pharmaceutical composition comprising said functional erythroid cells,
- wherein said isolated population of functional erythroid cells comprising said receiver polypeptide must be made by a method comprising the following steps:
- i introducing into a population of cultured or freshly isolated erythroid cell precursors an exogenous nucleic acid encoding said receiver polypeptide, wherein said receiver polypeptide comprises PAL;
- and
- ii culturing the population of erythroid cell precursors that contain said receiver polypeptide to provide the isolated population of functional erythroid cells comprising said receiver polypeptide.
3 more elements
Dependencies are inferred from the claim text.