Article

RNA Activation of CEBPA in PBMCs Enhances α-L-Iduronidase Expression A Translational Adjuvant Therapy for MPS I After Bone Marrow Transplantation

2025-08-21

Abstract excerpt

<title>Abstract</title> <p>Background Hurler syndrome, the most severe form of mucopolysaccharidosis type I (MPS I), is a rare genetic disorder caused by mutations in the IDUA gene, leading to a deficiency of the α-L-iduronidase enzyme. While current treatments offer some benefits, there remains a significant unmet medical need. We have identified a potential new therapeutic approach using MTL-CEBPA, a drug that...

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Literature Corpus work: be693fba-28c7-5f36-a012-ea9419c698c1
DOI: 10.21203/rs.3.rs-7346641/v1

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