Researchers at two U.S. universities have developed a method by which genetic diseases in utero can be treated via an injection into the surrounding fluid. They designed specific genetic sequences to alter the aberrant gene expressions in the fetus.
An interventionist approach such as an injection straight to the fetus can be dangerous and have developmental consequences. But delivering a drug to the amniotic fluid poses much less of a risk and has shown in mouse studied to be effective.
The scientists from Rosalind Franklin University and Oregon Health and Science University engineered short sequences of nucleic acids called antisense oligonucleotides that are designed to bind to specific genes. In the animal studies, when these were injected into the amniotic cavity, mice demonstrated alterations in their gene expression for up to a month after birth.
It’s the first of its kind that showed successful embryonic transfer with amniotic administration, according to a release.
“A major barrier to the development of treatments for congenital disorders is the risk to the developing fetus that interventions may pose,” lead author Michelle Hastings of RFU said. “Our demonstration that this promising type of therapeutic can be delivered to the amniotic cavity is an important advance for fetal treatment of disease.”