This was written by The ConversationRecently, two children were treated for Tay-Sachs disease. This treatment is associated with the use of an innovative method – gene therapy. Read more about the features of therapy and its results in the material. Discuss therapy” alt=”Tay-Sachs disease was first treated with gene therapy” Tay-Sachs disease was first treated with gene therapy />
Tay-Sachs syndrome, according to the author, is a severe neurological disease. It is caused by a deficiency of a special enzyme – HexA. In this case, one of the characteristic symptoms of such a disease is a red spot on the eye. The problem is that usually people with this diagnosis die by the age of five.
Scientists from the Chan School of Medicine at the Massachusetts Institute of Technology and Auburn University have created a new treatment for Tay's disease – Saxa. It lies in the fact that two harmless viral vectors are used in the course of therapy. They transmit DNA instructions to brain cells and thus “teach” them to produce the necessary element – HexA.
As for the results of treating children, they are positive. The first child was 2.5 years old at the time of treatment. Three months after the therapy, his muscle control improved and he was able to focus on objects. This child is now five years old and is in a stable condition, he has not had any seizures since the treatment.
The second child received treatment at seven months. Three months later, his brain began to develop much better. Now, at two years old, he also does not experience seizures.
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Source: The Conversation