Journal of mucopolysaccharidosis and rare diseases

  • 제2권1호
  • /
  • Pages.17-18
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  • 2016
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  • 2465-8936(pISSN)
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  • 2465-9452(eISSN)
뮤코다당증연구학회 (Association for Research of MPS and Rare Diseases)
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Development of New Strategies for Enzyme Replacement Therapy for Lysosomal Storage Disorders

  • Ko, Ah-Ra (Research Institute for Future Medicine, Samsung Biomedical Research Institute)
  • 투고 : 2016.05.30
  • 심사 : 2016.06.12
  • 발행 : 2016.06.30
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초록

Enzyme replacement therapy (ERT) is a well-established means of treating lysosomal storage disease (LSD). However, classical IV infusion based ERT method produces less than ideal results, especially, CNS defects and quality of life in patients. To improve these main problems of parental IV formulation for LSDs, we investigate modified ERT method and evaluated the efficacy in animal model.

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참고문헌

  1. Braulke T, Bonifacino JS. Sorting of lysosomal proteins. Biochim Biophys Acta 2009;1793:605-14. https://doi.org/10.1016/j.bbamcr.2008.10.016
  2. Al Sawaf S, Mayatepek E, Hoffmann B. Neurological findings in Hunter disease: pathology and possible therapeutic effects reviewed. J Inherit Metab Dis 2008;31:473-80. https://doi.org/10.1007/s10545-008-0878-x