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In Utero Gene Editing Could Prevent Hereditary Diseases
Currently, very few heritable diseases have gene therapies that have been approved by the FDA. One therapy, called Zolgensma, treats spinal muscular atrophy in newborns and children up to age two. But halting the disease in childhood may still be too late to avoid lifelong health issues. Scientists are now exploring the possibility of using in utero gene editing to prevent hereditary diseases and give babies a normal life from the start.
Research on In Utero Gene Editing
In 2019, Turkish researchers published evidence that in utero gene editing for spinal muscular atrophy could work in mice. Beltrán Borges, a postdoctoral researcher of pediatric surgery at the University of California, San Francisco, wanted to take this one step further and take it to sheep, which are well-studied subjects for this disease. Borges examined where the gene editing machinery would go if injected through either the umbilical vein or directly into the cranium.
Testing the two routes
Borges’s team tested the two routes by injecting a harmless virus carrying genetic instructions that would make the recipient cells glow green, indicating where they had landed. According to preliminary results Borges shared at the conference, the instructions sent by umbilical injections went where he hoped, like the brain, spinal cord, and muscle cells. But there was a catch: They also went where they shouldn’t.
The Risk of Germline Edits
Borges reported a small number of locations where genetic material entered female fetal lambs’ egg cells. It’s essential to avoid doing anything that might enable the editing of reproductive or “germline” cells, because those DNA changes could be passed down to the next generation. Gene replacement therapies, including this experiment, don’t edit an individual’s genome and should not be heritable.
The Role of Immunity
One of the other big challenges researchers are anticipating is immune response. Many people have antibodies for the Cas9 protein that Crispr uses to cut DNA, which means that their bodies may reject the therapy altogether. Maternal immunity does foil in utero gene therapy in some cases. When pregnant mice had immunity to the AAV9 virus often used to deliver gene therapy, more of their fetal offspring died due to the maternal immune response. Researchers are considering whether injecting the therapy directly into the umbilical cord early in pregnancy may safeguard the fetus from the mother’s immune response.
Challenges
It’s still very early days for gene therapy in the womb, and there are challenges to overcome such as drug delivery, immune rejection, and the risk of germline edits. It will take more research to ensure the safety of both fetus and parent, and to test whether the benefits of the therapy last long after treatment. “It is all very proof of concept,” says Borges, estimating that the first human in utero trials are probably still 5 to 10 years away.
Conclusion
In utero gene editing shows promise for preventing hereditary diseases, but there are still many challenges to overcome before human trials can begin. Researchers will need to ensure the safety of both fetus and parent, and establish the long-term efficacy of the therapy. However, early results are positive and offer hope for a future where hereditary diseases can be prevented from the start.
FAQ
What is in utero gene editing?
In utero gene editing is a form of gene therapy that involves editing a fetus’s genome while still in the womb. The goal is to prevent hereditary diseases and give babies a normal life from the start.
What are some challenges associated with in utero gene editing?
There are several challenges associated with in utero gene editing. Drug delivery, immune rejection, and the risk of germline edits are some potential risks that need to be addressed. Researchers will also need to ensure the safety of both fetus and parent, and establish the long-term efficacy of the therapy.
When are the first human in utero trials expected to begin?
Beltrán Borges, a postdoctoral researcher of pediatric surgery at the University of California, San Francisco, estimates that the first human in utero trials are probably still 5 to 10 years away.
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