Scientists say tinkering with genes could cure neurological diseases

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Brain editing is a concept that perhaps evokes B-movie rehashes of Mary Shelley’s “Frankenstein”: a catgut-stitched head flipped open like the lid of a trash can while subjected to the swivel-eyed probings of a lion-haired scalpel-wielder in a long white coat.

But a recent “spate of technological advances” could see something like brain editing become a reality. Recent breakthroughs in CRISPR-derived gene-editing therapies for blood, liver and eye diseases may herald similar treatments for neurological conditions, according to researchers speaking to science journal Nature.

Tests on mice have prompted optimism that scientists will soon be able to find a way to treat brain diseases or even figure out the mysterious origins of seizures, including such puzzles as whether or not these can be traced to genetic mutations.

Scientists at the Massachusetts Institute of Technology and Harvard University recently reported having used CRISPR to repair mutations in mice that, when seen in humans, lead to a condition called alternating hemiplegia of childhood (AHC), which typically causes toddlers to have seizures and sporadic paralysis. The team said they fixed mutations in the cortex, a brain region that controls learning and memory.

Meanwhile, a team of scientists based in Shanghai said they had carried out similar work targeting a mutation which has been linked to epilepsy when manifesting in humans.

“Gene-editing technology, which can rewrite small snippets of a cell’s genome, is ready to correct some of these mutations,” according to Nature, suggesting that human trials could potentially be “just a few years away.”

Neurological conditions are likely to be trickier to treat than those affecting other organs, however, not least because the brain is “surrounded by a defensive barrier that can prevent many substances from entering” – a hurdle not encountered when attempting to treat diseases related to other parts of the body.

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