Prenatal genetic therapy for SMA shows promise in a mouse study

Researchers have taken preliminary steps toward treating a devastating neurodevelopmental disorder, spinal muscular atrophy, with a genetic therapy delivered in the womb, before the worst damage to motor neurons can occur. 

The scientists at the University of California, San Francisco, and Johns Hopkins University in Baltimore injected an antisense oligonucleotide, or ASO, into the amniotic fluid of pregnant mice, and from there it traveled into the brains and spinal cords of mouse fetuses with the genetic disorder and substantially improved the motor function of the resulting pups. 

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