Gene Therapy gel
In the USA, a gel was created for the treatment of "butterfly disease"
Scientists from Stanford University (USA) have invented a gel that can help patients with "butterfly disease". Its official name is epidermolysis bullosa, according to Nature Medicine (Gurevich et al., In vivo topical gene therapy for recurrent dystrophic epidermolysis bullosa: a phase 1 and 2 trial).
With such a disease, the skin becomes extremely vulnerable and becomes covered with blisters and wounds at the slightest touch. Severe forms are considered incurable and can lead to death.
Previously, experts have tested several treatments for this genetic disease, including stem cell therapy and skin grafting. But they turned out to be long and expensive and were not suitable for everyone.
The authors of the new development have come up with a cheaper and simpler treatment. This is a topical gel that can be applied to the skin during dressings.
Patients with recessive dystrophic epidermolysis bullosa (RDEB) participated in laboratory tests. With this diagnosis, the cells lack the genetic instructions to create collagen VII. This protein anchors the basic structure of human skin, linking the dermis and epidermis.
The new method of therapy is based on the replacement of the defective C7 gene encoding collagen VII in patients. The gel "injects" genetically modified fibroblasts directly into the skin using the herpes virus (HSV-1).
This virus is able to infect skin cells and eludes the human immune system. Scientists changed it so that it could not reproduce and infect other parts of the body, and used it to "transport" two functional copies of the COL7A1 gene.
The tests showed that the patients' skin cells began to independently produce collagen VII nine days after treatment. For some, the beneficial effect remained for three months. However, then the collagen began to break down again. Scientists noted that the gel must be periodically reapplied.
Not too faint–hearted readers can see pictures of skin areas of one of the patients before and after treatment here - VM.
The gel did not cause side effects and had the same effect on patients of both sexes and all age groups. The scientists stressed that the wounds treated by him remained closed, and did not reappear, as in patients receiving a placebo.
"The therapy strengthens the skin and breaks the painful and destructive cycle of opening and closing wounds experienced by patients with epidermolysis bullosa," the scientists stressed.
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