MITOCHONDRIAL

DISEASE
Mitochondrial genetic

disorders
Mitochondrial diseases are long-term,
genetic, often inherited disorders that
occur when mitochondria fail to produce
enough energy for the body to function
properly. One in 5,000 individuals has a
genetic mitochondrial disease.
symptoms:
Poor growth
Loss of muscle coordination
Muscle weakness
Seizures
Autism
Problems with vision and/or hearing
Developmental delay

treatment:

The gene therapy treatment, known as the

mitochondrially targeted zinc finger-
nuclease, or mtZFN, recognises and then
eliminates the mutant mitochondrial DNA,
based on the DNA sequence differences
between healthy and mutant
mitochondrial DNA.