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Novel transferrin receptor-mediated enzyme replacement therapy efficiently treats myogenic and neurogenic aspects of Pompe disease in mice by Kelly George, Raquel Riley, Shan Zhou, Elizabeth Allen, Laurie Smith, Elena Kistanova, Sofia Kinton, Lilu Guo, Dinesh Bangari, Ayman Ismail, Jayaprakash Thummapudi, Fawzi Boumezbeur, Erwan Selingue, Dorothée Tamarelle, Cecile Capdevila, Tuan-Minh Do, Dominique Lesuisse, Nina Leksa, Pablo Sardi, Arjan van der Flier
Published 2025-09-01Get full text
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