Identification of a novel fusion Iduronidase with improved activity in the cardiovascular system

Sarah Kim,Michael J. Przybilla,Chester B. Whitley,Li Ou,Mahmoud Al-Kofahi,Jeanine R. Jarnes

Molecular Genetics and Metabolism Reports（2022）

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摘要

The addition of a six amino acid peptide improved iduronidase's activity levels in the heart and plasma. The short length of this LT peptide facilitates its use as fusion enzymes encoded as gene therapy or administered as enzyme replacement therapy. More broadly, the LT peptide may aid in developing therapies for numerous lysosomal diseases.

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关键词

Lysosomal diseases,Mucopolysaccharidosis type I,Fusion enzyme,Gene therapy,Pharmacology,Pharmacokinetics

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