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Articles

Intra-amniotic antisense oligonucleotide treatment improves phenotypes in preclinical models of spinal muscular atrophy

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By Becca A. Flitter, Huilan Wang, Stephen Brown, Wan-Jin Chen, Beltran Borges, Maria Clarke, Akos Herzeg, Joshua Ross, Lingling Kong, Amy K Smith, Tony Lum, Marco Bonilla Cordero, Nalin Gupta, Sarah Cook, Heather Murray, John Matson, Madeline Denton, Fareha Moulana Zada, Mandeep Singh
| Science Magazine Verified
Editor’s summary Treatment of spinal muscular atrophy (SMA) ideally starts in the early postnatal period, but patients with severe forms of SMA may still develop substantial neurological deficits despite this. Here, Borges and colleagues showed that prenatal treatment with antisense oligonucleotides (ASOs) via intra-amniotic injection improved outcomes in two mouse models of severe SMA.
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