MMUT-deficient zebrafish (methylmalonic acidemia)
CRISPR/Cas9-generated MMUT-deficient zebrafish show liver and kidney mitochondriopathy, behavioural abnormalities, increased mortality and oxidative stress.
模型身份
- 模型名称
- MMUT-deficient zebrafish (methylmalonic acidemia)
- 物种
- zebrafish
- 基因型／合子状态
- MMUT-deficient
生物学特性
- 适用研究问题
- What genetically engineered animal models exist for methylmalonic acidemia and what are their characteristics.
- 模型局限
- A whole-organism larval/juvenile model; metabolite handling and organ size differ from mammals, limiting direct pharmacokinetic extrapolation.