COMPOSITIONS, DEVICES AND METHODS FOR TREATING FABRY DISEASE

• Main Authors: MILLER, ROBERT JAMES, PAPAKOSTA, MARIANTHI, HEIDEBRECHT, RICHARD, SMITH, DEVYN MCKINLEY, SEWELL, JARED A, GONZALEZ, FRANCISCO CABALLERO, PERITT, DAVID, BEAUREGARD, MICHAEL, VEISEH, OMID, WAY, JEFFREY CHARLES, YIN, ZOE, BARNEY, LAUREN EMILY, JOHNSTON, ERIKA ELLEN, WOTTON, PAUL KEVIN, O'CONNOR, OWEN, MAKINO, ELINA, OBERLI, MATTHIAS ALEXANDER, CARMONA, GUILLAUME, FLUHARTY, BRIAN RICHARD
• Format: Patent
• Language: English; French
• Published: 01.10.2020
• Subjects: 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

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. L'invention concerne des cellules RPE modifiées pour sécréter une protéine GLA, ainsi que des compositions, des préparations pharmaceutiques et des dispositifs implantables comprenant les cellules RPE modifiées, et des méthodes de fabrication et d'utilisation de celles-ci pour le traitement de la maladie de Fabry.