Overview

• UCSF-led research demonstrated that prenatal delivery of antisense oligonucleotides (ASOs) into the amniotic fluid prevents motor neuron degeneration in spinal muscular atrophy (SMA) animal models.
• Mouse studies showed significant improvements in survival, motor function, and spinal cord neuron preservation compared to postnatal treatment or no treatment.
• Safety and biodistribution studies in sheep confirmed that ASOs reached key fetal organs without causing toxicity or developmental harm.
• Researchers plan to apply for FDA approval to initiate human clinical trials, marking a critical step toward translating this approach to the clinic.
• The minimally invasive delivery method, described as an 'inverse amniocentesis,' could potentially be adapted for other severe genetic disorders identified during pregnancy.