کد مقاله کد نشریه سال انتشار مقاله انگلیسی نسخه تمام متن
2839298 1165106 2006 8 صفحه PDF دانلود رایگان
عنوان انگلیسی مقاله ISI
Utrophin upregulation for treating Duchenne or Becker muscular dystrophy: how close are we?
موضوعات مرتبط
علوم زیستی و بیوفناوری بیوشیمی، ژنتیک و زیست شناسی مولکولی پزشکی مولکولی
پیش نمایش صفحه اول مقاله
Utrophin upregulation for treating Duchenne or Becker muscular dystrophy: how close are we?
چکیده انگلیسی

Duchenne muscular dystrophy (DMD) is a severe muscle-wasting disorder for which there is currently no effective treatment. This disorder is caused by mutations or deletions in the gene encoding dystrophin that prevent expression of dystrophin at the sarcolemma. A promising pharmacological treatment for DMD aims to increase levels of utrophin, a homolog of dystrophin, in muscle fibers of affected patients to compensate for the absence of dystrophin. Here, we review recent developments in our understanding of the regulatory pathways that govern utrophin expression, and highlight studies that have used activators of these pathways to alleviate the dystrophic symptoms in DMD animal models. The results of these preclinical studies are promising and bring us closer to implementing appropriate utrophin-based drug therapies for DMD patients.

ناشر
Database: Elsevier - ScienceDirect (ساینس دایرکت)
Journal: - Volume 12, Issue 3, March 2006, Pages 122–129
نویسندگان
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