Evidence is mounting that clinicians can treat serious genetic disorders prenatally by injecting medicine into the amniotic fluid, thus preventing damage that begins in utero.
A study lead by Tippi MacKenzie, MD, Co-director of the Center for Maternal Fetal Precision Medicine, found that delivering a genetic therapy for spinal muscular atrophy (SMA) via the amniotic fluid was safe and helped prevent damage to motor neurons in the spinal cord, a part of the central nervous system that is responsible for movement. One experiment was done in mice with SMA — a neurodegenerative disease that causes muscular weakness, atrophy, and death if untreated. Another was done in sheep that did not have the disease to demonstrate that the method is safe… Read the full UCSF News article by Jess Berthold: A Step Forward in Treating Serious Genetic Disorders Before Birth
Research published in Science Translational Medicine, 14 May 2025, 17(798): Intra-amniotic antisense oligonucleotide treatment improves phenotypes in preclinical models of spinal muscular atrophy
Related coverage:
STAT: Prenatal genetic therapy for SMA shows promise in a mouse study
Inside Precision Medicine: Prenatal RNA therapy could prevent irreversible damage in spinal muscular atrophy
