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MMUT-deficient zebrafish (methylmalonic acidemia)

CRISPR/Cas9-generated MMUT-deficient zebrafish show liver and kidney mitochondriopathy, behavioural abnormalities, increased mortality and oxidative stress.

Model identity

Model name
MMUT-deficient zebrafish (methylmalonic acidemia)
Species
zebrafish
Genotype / zygosity
MMUT-deficient

Biological characteristics

Research questions
What genetically engineered animal models exist for methylmalonic acidemia and what are their characteristics.
Limitations
A whole-organism larval/juvenile model; metabolite handling and organ size differ from mammals, limiting direct pharmacokinetic extrapolation.

References

  1. Genetically engineered animal models of methylmalonic acidemia review (PMID:41239408)
  2. ARRIVE 2.0 guidelines