A Better Way to Deliver Fetal Therapy for Serious Genetic Disorders

In a discovery that opens the door to a less invasive way of treating some serious disorders before birth, UC San Francisco scientists and colleagues have found that delivering medicine through amniotic fluid is as effective as delivering it to the fetal brain via cerebrospinal fluid. The experiment was done in mice with a model of the human genetic disorder called Angelman syndrome. Treating genetic diseases like Angelman in utero could prevent serious symptoms that begin while the fetus is still developing. It’s also easier to access neurons in the fetal brain because the blood-brain barrier that normally acts as a filter to prevent molecules from reaching the brain is not yet fully formed. The treatment uses therapeutic molecules called antisense oligonucleotides, or ASOs, that can alter the expression of genes through interactions with RNA, which creates proteins.  
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