Treatment strategy for hematopoietic stem cell transplantation as a curative therapy for mucolipidosis type III and development of gene therapy as a novel treatment approach

Molecular Genetics and Metabolism 2026

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Authors: Hiroaki To, Yasuyuki Fukuhara, Hirotoshi Sakaguchi, Kotaro Sakaya, Tetsumin So, Norio Sakai, Kenji Kurosawa, Motomichi Kosuga

DOI: 10.1016/j.ymgme.2025.109645

Cited by: 0

Fields of Study & Subjects

Lysosomal Storage Disorders Research Glycogen Storage Diseases and Myoclonus Biomedical Research and Pathophysiology