Deadly brain disease could be treated in the womb with gene therapy

PRinc photo of fetus at weeks

A research team from the UK and Singapore showed that a neurodegenerative condition called Gaucher disease, which can be fatal, can be prevented in mice by treating fetuses with gene therapy.

The study, published in Nature Medicine, showed mice fetuses with Gaucher disease that received an injection of the gene therapy were able to express the missing enzyme. Furthermore, animals treated with the gene therapy lived for four and a half months after birth compared to 15 days for untreated mice.

While the findings underscore the potential of using gene therapy to prevent and cure fetal diseases in humans in utero, the approach is still a long way from being tested in humans.

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Read full, original post: Gene Therapy Could Prevent Deadly Brain Disease

Related article:  Application for pricey gene therapy Zolgensma contained manipulated data, but drug will remain on market, FDA says

 

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