Compositions, devices and methods for treating Fabry disease

Described herein are RPE cells engineered to secrete a GLA protein, as well as compositions, pharmaceutical preparations, and implantable devices comprising the engineered RPE cells, and methods of making and using the same for treating Fabry disease.

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Bibliographische Detailangaben
Hauptverfasser:	CARMONA, Guillaume, JOHNSTON, Erika Ellen, WOTTON, Paul Kevin, OBERLI, Matthias Alexander, YIN, Zoe, HEIDEBRECHT, Richard, WAY, Jeffrey Charles, VEISEH, Omid, PAPAKOSTA, Marianthi, MAKINO, Elina, O'CONNOR, Owen, SEWELL, Jared A, BEAUREGARD, Michael, MILLER, Robert James, PERITT, David, SMITH, Devyn McKinley, FLUHARTY, Brian Richard, BARNEY, Lauren Emily, GONZALEZ, Francisco Caballero
Format:	Patent
Sprache:	eng
Schlagworte:	CHEMICAL ASPECTS OF BANDAGES, DRESSINGS, ABSORBENT PADS, ORSURGICAL ARTICLES DISINFECTION, STERILISATION, OR DEODORISATION OF AIR HUMAN NECESSITIES HYGIENE MATERIALS FOR BANDAGES, DRESSINGS, ABSORBENT PADS, OR SURGICALARTICLES MEDICAL OR VETERINARY SCIENCE METHODS OR APPARATUS FOR STERILISING MATERIALS OR OBJECTS INGENERAL PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
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Beschreibung
Zusammenfassung:	Described herein are RPE cells engineered to secrete a GLA protein, as well as compositions, pharmaceutical preparations, and implantable devices comprising the engineered RPE cells, and methods of making and using the same for treating Fabry disease.