"Genie" solution for a disease that kills 200 thousand children annually by seamless gene correction of β-thalassemia mutations

A team of Chinese researchers have corrected a genetic defect in human embryos with a serious hereditary blood disease, thalassemia.
Thalassemia is a genetic disease that prevents the proper synthesis of hemoglobin, a substance found in the erythrocyte that allows the transport of oxygen between organs.
The disease causes the blood poor to be fatal in some of its most serious forms, and is related to a local change in a gene called HPP.
Using a new technique for genetic modification based on chemically altered RNA, researchers at Sun Yat-sen University in Canton have corrected one form of this change on human embryos that are not viable.
The study, published by the magazine "Protein and Cell" specialist that the technology used is based on the method of
"Crispr / Cas 9" but with the addition of the use of Enzyme allowed accurate correction of the position of transformation in DNA.
This method succeeded in a case of every five.
About 200,000 children are born each year with thalassemia, making it the most common genetic disease. It is particularly present in the populations of the Mediterranean countries and in Asia.

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