Title of article
Treatment of lysosomal storage disorders: Focus on the neuronal ceroid-lipofuscinoses
Author/Authors
Chris Pierret، نويسنده , , Jason A. Morrison، نويسنده , , Mark D. Kirk، نويسنده ,
Issue Information
روزنامه با شماره پیاپی سال 2008
Pages
14
From page
429
To page
442
Abstract
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 pharma ceutical 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.
Keywords
neuronal ceroid-lipofuscinoses , lysosomal storage , enzyme replacement , Stem cell , gene therapy , substrate reduction
Journal title
Acta Neurobiologiae Experimentalis
Serial Year
2008
Journal title
Acta Neurobiologiae Experimentalis
Record number
672743
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