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CRISPR injection into the brain of the embryo can cure genetic disorder

Quite a controversial new studysuggests that there may be a way to prevent Angelman syndrome, a genetic disorder that can cause seizures, difficulties in communication and is associated with diseases of the autism spectrum. The fact is that in order to test the experimental treatment in mice, scientists had to inject CRISPR enzymes, which edit the gene, directly into the brain of the developing fetus. Genetically modified fruits are uncharted ethical territory. A potentially useful experimental treatment should be balanced by anxiety about the patient's well-being.

As always, on mice

The treatment that was presented at the scientificconference in February, but not yet in academic journals, affects a specific UBE3A gene, which helps cells break down and process proteins.

When this gene mutates in the developing brain,Angelman syndrome occurs. But research shows that it can be avoided if the genes turn on before birth. In an experiment with 10 embryonic mice, scientists applied CRISPR to reactivate the gene and found that editing worked on half of the neurons of the brain of each mouse by the time they were five months old. Editing had a similar effect on human grown neurons.

"The sooner you return the genes and try to solvethe problem is, the better the therapeutic outcome will be, ”says Mark Zilka of the University of North Carolina at Chapel Hill, head of research. "There is a great interest in trying to break through this barrier and get the opportunity for prenatal treatment."

Zilka told Spectrum that he might tryto conduct an experiment on humans for four years, but other scientists are less pleased with this prospect, since it implies that they have to crack the genes of babies, much like what the Chinese scientist He Jiankui did.

"Any manipulation of the genes in the fetal brain is dangerous, so I have concerns," said oncologist Antonio Bedalov of the Fred Hutchinson Cancer Research Center.

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