Adeno-associated viral gene therapy for mucopolysaccharidoses exhibiting neurodegeneration

Research output: Contribution to journalReview article

5 Citations (Scopus)

Abstract

The mucopolysaccharidoses (MPS) are a subgroup of lysosomal storage disorders that are caused by mutations in the genes involved in glycosaminoglycan breakdown. Multiple organs and tissues are affected, including the central nervous system. At present, hematopoietic stem cell transplantation and enzyme replacement therapies are approved for some of the (non-neurological) MPS. Treatments that effectively ameliorate the neurological aspects of the disease are being assessed in clinical trials. This review will focus on the recent outcomes and planned viral vector-mediated gene therapy clinical trials, and the pre-clinical data that supported these studies, for MPS-I (Hurler/Scheie syndrome), MPS-II (Hunter syndrome), and MPS-IIIA and -IIIB (Sanfilippo syndrome).

Original languageEnglish
Pages (from-to)1043-1052
Number of pages10
JournalJournal of Molecular Medicine
Volume95
Issue number10
DOIs
Publication statusPublished - Oct 2017
Externally publishedYes

Keywords

  • Central nervous system
  • Gene therapy
  • Lysosomal storage disease
  • Mucopolysaccharidosis
  • Viral vectors

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