Modelling the neuropathology of lysosomal storage disorders through disease-specific human induced pluripotent stem cells

• Main Authors: Julianna Kobolák, Kinga Molnár, Eszter Varga, István Bock, Bálint Jezsó, Annamária Téglási, Shuling Zhou, Maria Lo Giudice, Marianne Hoogeveen-Westerveld, WWM Pim Pijnappel, Phetcharat Phanthong, Norbert Varga, Narisorn Kitiyanant, Kristine Freude, Hideyuki Nakanishi, Lajos László, Poul Hyttel, András Dinnyés
• Other Authors: BioTalentum Ltd.
• Format: Article
• Published: 2020
• Subjects: Biochemistry, Genetics and Molecular Biology
• Online Access: https://repository.li.mahidol.ac.th/handle/123456789/50127
• Institution: Mahidol University

© 2019 The Authors Mucopolysaccharidosis II (MPS II) is a lysosomal storage disorder (LSD), caused by iduronate 2-sulphatase (IDS) enzyme dysfunction. The neuropathology of the disease is not well understood, although the neural symptoms are currently incurable. MPS II-patient derived iPSC lines wer...