Treatment of lysosomal storage disorders: Focus on the neuronal ceroid-lipofuscinoses

• Autorzy: Pierret C. , Morrison J. A. , Kirk M. D.
• Języki publikacji: EN

Abstrakty

EN

Recent advances in our understanding of lysosomal storage disorders (LSDs) may lead to new therapies to treat the neuronal ceroid-lipofuscinoses (NCLs). In this review, enzyme replacement therapy, gene therapy, cell-mediated therapy and pharmaceutical treatments are considered across the LSDs and extended to therapies for the NCLs. It is likely that a combination of approaches will produce the most beneficial clinical outcome for treatment of pathologies displayed by the NCLs.

• Wydawca: -
• Czasopismo: Acta Neurobiologiae Experimentalis
• Rocznik: 2008
• Tom: 68
• Numer: 3
• Opis fizyczny: p.429-442,ref.

Twórcy

autor

Pierret C.

• Division of Biological Sciences, University of Missouri, Columbia, MO 65211, USA

autor

Morrison J. A.

• Division of Biological Sciences, University of Missouri, Columbia, MO 65211, USA

autor

Kirk M. D.

• Division of Biological Sciences, University of Missouri, Columbia, MO 65211, USA

Uwagi

PL

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