# Animal models of methylmalonic acidemia: insights and challenges

## TL;DR

This review discusses animal models used to study methylmalonic acidemia, a rare genetic disorder, and their role in understanding the disease and developing treatments.

## Contribution

The paper provides a comprehensive overview of genetically engineered animal models for methylmalonic acidemia and their implications for future therapies.

## Key findings

- Murine and zebrafish models offer insights into MMA pathogenesis and treatment evaluation.
- Genetically engineered models have limitations that need addressing for clinical translation.
- Integration of model insights can guide future therapeutic innovations.

## 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.