Academics

Stanford Researchers Use Gene Therapy To Cure Rare Skin Disease

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Epidermolysis Bullosa or EB is a very rare skin disease characterized by the blistering and falling off of the skin even with the slightest touch. It is very rare, with only 1 out of 50,000 children, that s lot of people have never heard about it. Yet, those who suffer from it undergo the most severe pain which has no cure other than putting some bandages around it. However, researchers at Stanford University are offering hope to EB patients through gene therapy.

Those who are suffering from epidermolysis bullosa suffers from the slightest closest contact. It afflicts the person with so much pain that most of the time they wish they were dead. In fact, in the Netherlands, two children have been euthanized to stop their suffering from EB.

EB is a result of the lack of type-7 collagen in the body. This type of collagen is what fuses the lower and the upper skin together. Due to unknown reasons, however, some sort of DNA mutation blocks the production of type-7 collagen.

However, with the team of researchers at Stanford conducting a gene-therapy study to treat EB, this disease will soon have a cure if the stages are successful. What the scientists did was get a part of the patient's skin and genetically modify them into card-sized measurements. Once the skin is modified, it is then re-attached to the skin to cover the injuries.

The team has already treated six adults so far and will soon start accepting teenagers and kids. They also said that those who have received the treatment are likely to receive treatment every two or three years.

Aside from the study in treating EB, other gene therapy studies are conducted to treat other rare and inherited diseases, such as hemophilia and blindness. The greatest challenge, researchers say, is delivering the right genetic material to the right place of the body.

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