Scholarlink

<p>Glycogen storage disease type III (GSDIII) is a rare genetic disorder leading to abnormal glycogen storage in the liver and skeletal muscle. In this study, we conducted a comparative gene expression analysis of several in vitro and in vivo models and identified galectin-3 as a potential biomarker of the disease. Interestingly, we also observed a significant decrease in galectin-3 expression in mice treated with an AAV gene therapy. Finally, galectin-3 expression was studied in muscle biopsies of GSDIII patients, confirming its increase in patient tissue. Beyond the identification of this novel biomarker, our study offers a new perspective for future therapeutic developments.</p>

错误：搜索内容不能为空，请输入英文关键词
错误：关键词超出字数限制，请精简
高级检索

Galectin-3: a novel biomarker of glycogen storage disease type III

  • 期刊论文

  • 出版模式：

    开放获取

  • 发表日期：

    2025年4月14日

查看全文
  • Lucille Rossiaud,
  • Quentin Miagoux,
  • Manon Benabides,
  • Océane Reiss,
  • Louisa Jauze,
  • Margot Jarrige,
  • Hélène Polvèche,
  • Edoardo Malfatti,
  • Pascal Laforêt,
  • Giuseppe Ronzitti,
  • Xavier Nissan,
  • Lucile Hoch

摘要

Glycogen storage disease type III (GSDIII) is a rare genetic disorder leading to abnormal glycogen storage in the liver and skeletal muscle. In this study, we conducted a comparative gene expression analysis of several in vitro and in vivo models and identified galectin-3 as a potential biomarker of the disease. Interestingly, we also observed a significant decrease in galectin-3 expression in mice treated with an AAV gene therapy. Finally, galectin-3 expression was studied in muscle biopsies of GSDIII patients, confirming its increase in patient tissue. Beyond the identification of this novel biomarker, our study offers a new perspective for future therapeutic developments.