کد مقاله کد نشریه سال انتشار مقاله انگلیسی نسخه تمام متن
1998224 1065764 2014 6 صفحه PDF دانلود رایگان
عنوان انگلیسی مقاله ISI
Enhanced brain distribution of modified aspartoacylase
ترجمه فارسی عنوان
توزیع مغز پیشرفته آسپارتاکیلاز اصلاح شده
موضوعات مرتبط
علوم زیستی و بیوفناوری بیوشیمی، ژنتیک و زیست شناسی مولکولی زیست شیمی
چکیده انگلیسی

Canavan disease is a fatal neurological disorder caused by defects in the gene that produces the enzyme aspartoacylase. Enzyme replacement therapy can potentially be used to overcome these defects if a stable enzyme form that can gain access to the appropriate neural cells can be produced. Achieving the proper cellular targeting requires a modified form of aspartoacylase that can traverse the blood–brain barrier. A PEGylated form of aspartoacylase that shows dramatic enhancement in brain tissue access and distribution has been produced. While the mechanism of transport has not yet been established, this modified enzyme is significantly less immunogenic than unmodified aspartoacylase. These improved properties set the stage for more extensive enzyme replacement trials as a possible treatment strategy.

ناشر
Database: Elsevier - ScienceDirect (ساینس دایرکت)
Journal: Molecular Genetics and Metabolism - Volume 113, Issue 3, November 2014, Pages 219–224
نویسندگان
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