α-L-iduronidase therapy for mucopolysaccharidosis type I

Research output: Contribution to journalReview article

13 Scopus citations

Abstract

More than 500 patients with mucopolysaccharidosis type IH (MPS IH; Hurler syndrome) have been treated with hematopoietic cell transplantation (HCT) throughout the world since the introduction of transplantation as therapy almost 30 years ago. More recently, the availability of recombinant α-L-iduronidase (IDUA) has resulted in the widespread treatment of less severe forms of MPS I with enzyme replacement therapy (ERT). In addition, over 50 MPS IH patients have been treated with a combination of ERT and HCT. The rationale for both ERT and HCT stems from the pivotal experiments performed 4 decades ago that showed α-L-iduronidase supplied in the environment can correct the accumulation of substrate in MPS I cells. Our purpose is to address the multiple applications associated with the therapeutic delivery of IDUA: intermittent delivery of recombinant protein (ERT), continuous administration through cellular therapy (HCT), the use of other stem cells or, potentially, correction of the enzyme defect itself through gene therapy approaches. Even though gene therapy and non-hematopoietic stem cell approaches, have yet to be tested in a clinical setting, it is possible that all these approaches will in the near future be a part of a paradigm shift from unimodal to multimodal therapy for MPS I.

Original languageEnglish (US)
Pages (from-to)743-751
Number of pages9
JournalBiologics: Targets and Therapy
Volume2
Issue number4
DOIs
StatePublished - 2008

Keywords

  • Co-modality therapy
  • Enzyme replacement therapy
  • Hematopoietic cell transplantation
  • Hurler syndrome
  • Mucopolysaccharidosis type I

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