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Chinese scientists develop gene-editing tool to target incurable diseases

Mitochondrial DNA base editors could lead to gene therapies for serious conditions that currently lack curative therapies, team says

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Some human cells can have 1,000 to 2,000 mitochondria, each with their own copies of DNA. Mutations in mitochondrial DNA are responsible for several serious diseases in humans. Photo: Shutterstock
Victoria Bela
Chinese researchers have made a major advance in the science of gene editing by developing a new way to treat diseases that cannot be cured through existing methods.
Their new gene editing tools, known as mitochondrial DNA base editors, can fix errors in the DNA inside a cell’s mitochondria. The scientists used these editors to create rats with diseases caused by these mitochondrial DNA errors and succeeded in making the first direct corrections of these flaws in animals.
These mitochondrial DNA base editors could lead to gene therapies for serious conditions that currently lack curative therapies, according to the team led by researchers from East China Normal University and Lingang Laboratory.
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“Efficient generation and correction of mutations in mitochondrial DNA (mtDNA) is challenging,” the researchers said in the first of two papers published in the peer-reviewed journal Nature Biotechnology on June 3.

Mitochondrial DNA mutations have been associated with several genetic diseases in humans. These include Leber hereditary optic neuropathy, which causes vision loss; Leigh syndrome, a neurological disorder; and mitochondrial encephalomyopathy, which can cause stroke-like episodes and muscle weakness.

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There is currently no cure for mtDNA diseases, which affect around one in 5,000 people worldwide and can lead to serious symptoms and premature death.

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