Gene therapy for Mucopolysaccharidoses.
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ABSTRACT:
SUBMITTER: Sawamoto K
PROVIDER: S-EPMC5986190 | biostudies-literature | 2018 Feb
REPOSITORIES: biostudies-literature
Publications
Gene therapy for Mucopolysaccharidoses.
Sawamoto Kazuki K Chen Hui-Hsuan HH Alméciga-Díaz Carlos J CJ Mason Robert W RW Tomatsu Shunji S
Molecular genetics and metabolism 20171226 2
Mucopolysaccharidoses (MPS) are a group of lysosomal storage disorders (LSDs) caused by a deficiency of lysosomal enzymes, leading to a wide range of various clinical symptoms depending upon the type of MPS or its severity. Enzyme replacement therapy (ERT), hematopoietic stem cell transplantation (HSCT), substrate reduction therapy (SRT), and various surgical procedures are currently available for patients with MPS. However, there is no curative treatment for this group of disorders. Gene therap ...[more]
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