Article
Intra-amniotic antisense oligonucleotide treatment improves phenotypes in preclinical models of spinal muscular atrophy.
Science translational medicine - 14 May 2025
Borges Beltran, Brown Stephen M, Chen Wan-Jin, Clarke Maria T, Herzeg Akos, Park Jae Hong, Ross Joshua, Kong Lingling, Denton Madeline, Smith Amy K, Lum Tony, Zada Fareha Moulana, Cordero Marco, Gupta Nalin, Cook Sarah E, Murray Heather, Matson John, Klein Stephanie, Bennett C Frank, Krainer Adrian R, MacKenzie Tippi C, Sumner Charlotte J
Abstract excerpt
Neurological disorders with onset before or at birth are a leading cause of morbidity and mortality in infants and children. Prenatal treatment has the potential to reduce or prevent irreversible neuronal loss and facilitate normal neurodevelopment. We hypothesized that antisense oligonucleotides (ASOs) delivered to the amniotic fluid by intra-amniotic (IA) injection could safely distribute to the fetal central...
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