Gene-Editing Experiment in Humans: We Have Some Good First Results


It’s true that genetic disorder affects the lives of about millions of people from everywhere. Doctors from around the globe hope to change this fact – they want to go inside the body of humans to alter and get rid of the disorders from the human DNA. A first experiment happened, and the early results show some promising preliminary results in treating rare disorders.

The University of North Carolina has a team behind who worked on a research about editing the genome of those people who suffer from the rare disease called the Hunter syndrome. They used a mouse model for the developing of therapy for this rare disorder – this happened 20 years ago. Being able to do this on a human being is actually amazing.

Does this treatment really work?

The first results are actually breath-taking. After four months of getting a medium dose of the treatment, urine samples of 2 patients displayed that the sugar compounds which are the markers of the Hunter syndrome have decreased by half. However, the team behind the research is not sure whether the treatment is working or not, but the fact is that the first results are good. They need more time to figure out how the immune system gets along with the doses. One of the team members stated that the base of these first results show that they can continue doing their work.

There are six people that have the Hunter syndrome (which is caused by the lack of a gene that helps the enzyme to break down the complex molecules of sugar). If not destroyed, these molecules can build up and make chaos in the whole body.

Among the symptoms, we can find heart diseases, joint stiffness and some severe cases of cognitive impairment.

The gene therapy can have the potential to improve the quality of those who fight the Hunter syndrome.


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