Injection of CRISPR in the fetal brain can cure a genetic disorder

Quite a controversial new study suggests there may be a way to prevent Angelman syndrome, a genetic disorder that can cause seizures, difficulties in communication associated with autism spectrum disorders. The fact that in order to test the experimental treatment on mice, the scientists had to enter the enzymes called CRISPR, that editing the gene directly into the brain of the developing fetus. Genetically modified fruit is uncharted ethical territory. Potentially useful experimental treatment should be balanced by concern about the welfare of the patient.

As always, in mice

The treatment, which was presented at a scientific conference in February, but hasn’t come out in academic journals, mentions specific gene UBE3A, which helps cells to break down and recycle proteins.

When this gene is mutated in the developing brain, there is a syndrome of Angelman. But studies show that it can be avoided if the genes are activated before birth. In the experiment with 10 embryonic mice, scientists have used CRISPR to reactivation of the gene and found that the edit worked half of the neurons in the brain of each mouse by the time they turned five months. Similar effect the edit has had on neurons grown man.

“The sooner you return the genes and try to solve the problem, the better the therapeutic outcome,” says mark zylka from the University of North Carolina at chapel hill, Director of research. “There is great interest to try to break through this barrier and to the possibility of prenatal treatment.”

Zilka told Spectrum that may try to experiment on humans for four years, but other scientists are less than happy with such a prospect, because it implies that you have to crack the code of babies, like what did Chinese scholar he Cisangkuy.

“Any tampering with the genes in the brain of the fetus is dangerous, so I have concerns,” says oncologist Antonio Bedalov from the cancer research Center Fred Hutchinson.