British researchers have developed a gene therapy which shows promise for treating patients with mitochondrial disease, it was announced last night.
The therapy has been successfully tested on mice, leading to “significant modification” to mitochondrial DNA, developers at Cambridge University say.
Current strategies to tackle the diseases focus on replacement therapy at the time of pregnancy and have been pioneered in Newcastle, UK. But the Cambridge team says this will prevent only a small number of cases.
The new treatment aims to identify and destroy mutant DNA. The developers say this leads to replacement with healthy copies, reducing the burden from cells with mutated DNA. The treatment is known as mitochondrially targeted zinc finger-nuclease.
The developers are hoping to move on to clinical trials.
They say it is the first-time programmable genome engineering tools have been used inside a living animal.
Researcher Dr Michal Minczuk said: “One idea for treating these devastating diseases is to reduce the amount of mutated mitochondrial DNA by selectively destroying the mutated DNA and allowing healthy DNA to take its place.”
Payam A. Gammage et al. Genome editing in mitochondria corrects a pathogenic mtDNA mutation in vivo. Nature Medicine 24 September 2018; doi: 10.1038/s41591-018-0165-9
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