This week, scientists reported that they were able to treat a serious genetic disorder in the womb — in mice. It sounds like science fiction, but this type of treatment may not be far away for humans. What will we need to figure out before we get there?
For the study published in Nature Medicine, scientists studied the neurodegenerative disorder Gaucher syndrome. Gaucher is caused by mutations in the GBA gene and leads to serious joint pain, convulsions, and cognitive impairment. To treat this, the researchers injected the fetus with a virus engineered to carry working (non-mutated) copies of the GBA gene to replace the mutated ones.
It wasn’t a perfect fix. The mice lived for 130 days instead of five days, but they did have some signs...
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