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<p>Methylmalonic acidemia (MMA) is a rare genetic disorder caused by disruptions in the metabolism of methylmalonic acid, resulting in severe neurological and systemic complications. Animal models have become indispensable in advancing our understanding of MMA pathogenesis, evaluating treatment options, and exploring new therapies. This review highlights recent advancements in MMA research, focusing on the characteristics, advantages, and limitations of various genetically engineered animal models, including murine and zebrafish models. By integrating insights from these models, this work aims to provide a foundation for future clinical applications and therapeutic innovations.</p><p><?noindent??><b>Clinical trial number</b> Not applicable.</p>

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Animal models of methylmalonic acidemia: insights and challenges

  • 期刊论文

  • 出版模式：

    开放获取

  • 发表日期：

    2025年11月14日

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  • Shan Shan,
  • Min Liu,
  • Yue Ma,
  • Meng Sun,
  • Yue Wang,
  • Hui Zou

摘要

Methylmalonic acidemia (MMA) is a rare genetic disorder caused by disruptions in the metabolism of methylmalonic acid, resulting in severe neurological and systemic complications. Animal models have become indispensable in advancing our understanding of MMA pathogenesis, evaluating treatment options, and exploring new therapies. This review highlights recent advancements in MMA research, focusing on the characteristics, advantages, and limitations of various genetically engineered animal models, including murine and zebrafish models. By integrating insights from these models, this work aims to provide a foundation for future clinical applications and therapeutic innovations.

Clinical trial number Not applicable.