Injection CRISPR in the fetal brain can cure a genetic disorder

Quite a controversial new study suggests that there may be a way to prevent the Angelman syndrome, a genetic disorder that can cause seizures, difficulty in communication and is associated with autism spectrum disorders. The fact is that in order to test an experimental treatment on mice, the scientists had to introduce enzymes CRISPR, editing the gene directly into the brain of the developing fetus. Genetically modified fruits - is uncharted ethical territory. Potentially useful experimental treatment must be balanced by a concern for the patient's well-being.

Injection CRISPR in the fetal brain can cure a genetic disorder

As always, mice

The treatment, which was presented at a scientific conference in February, but has not yet got into the academic journals, the UBE3A affects a specific gene, which helps to break down and recycle proteins cells.

When this gene is mutated in the developing brain, Angelman syndrome occurs. But studies show that it can be avoided if the genes be switched before the birth. In an experiment with 10 mice, scientists used embryonic CRISPR to reactivate the gene and found that editing has worked on half of each mouse brain neurons to the point where it was five months. Had similar effects editing and human neurons grown. "The sooner you return genes and try to solve the problem, the better therapeutic result," says Mark Tsilka University of North Carolina at Chapel Hill, who led the study. "There is great interest in, to try to break through this barrier and have prenatal treatment".

Tsilka told Spectrum, it may try to carry out an experiment on humans for four years, but other scientists have less pleased at the prospect, since it means that you have to crack babies genes, similar to that made Chinese scientist Hye Tszyankuy.

"Any manipulation of genes in the brain of the fetus - it is dangerous, so I have the fear," says oncologist Antonio Bedalov of the Cancer Research Center Fred Hutchinson.

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