Maria Praggastis

Maria Praggastis

Company: Pharmaceuticals

Job title: Staff Scientist Regeneron


TFRC-Targeted GAA Delivered as Gene Therapy Treats CNS & Muscle in Pompe Disease Model Mice 3:40 pm

Design and selection of anti-TFRC antibody fused to GAA Delivery of anti-TFRC:GAA as a liver depot gene therapy in Pompe disease Gaa-/- mice results in targeted uptake of GAA protein in need-to-treat tissues, including key cell types in the brain Anti-TFRC:GAA rescues glycogen storage in CNS, heart, and skeletal muscle in Gaa-/- miceRead more

day: Day One

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