Production and characterisation of a functional recombinant human palmitoyl-protein thioesterase 1 in K. phaffii for the treatment of CLN1

Molecular Genetics and Metabolism 2026

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Authors: Martyna Kasprzyk, Matthias Dierick, Stephen E. Meek, Tom Burdon, Eve V. Miller-Hodges, Thomas M. Wishart, Giovanni Stracquadanio

DOI: 10.1016/j.ymgme.2025.109495

Cited by: 0

Fields of Study & Subjects

Lysosomal Storage Disorders Research Metabolism and Genetic Disorders Glycogen Storage Diseases and Myoclonus