Animal models of methylmalonic acidemia: insights and challenges
Abstract
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.
How strong is the evidence?
Evidence Hub reads this as a review, done in animal, not in people. Its evidence level is 5 of 5 (Very low · case / narrative / animal / in vitro / in silico). Case reports, narrative reviews, and animal, cell or computer-model studies give the weakest evidence for human health.
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