MRC Mitochondrial Biology Unit
Mitochondria, often referred to as the ‘powerhouse of the cell’, contain their own DNA (mtDNA), which is susceptible to mutations. These mutations constitute a common cause of inherited neurological disorders. However, curative treatments remain elusive due to the challenges associated with editing the mitochondrial genome. While CRISPR/Cas9 is ineffective in targeting mtDNA, the newly developed base editors known as DdCBEs made this possible. In Lucia’s research, DdCBEs are used to create accurate in vivo models of mtDNA disease, with the aim of developing translational gene therapies aimed at preventing the neuronal dysfunction caused by mitochondrial mutations.
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