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Transgenic Stem Cells Treat Patient with Severe Genetic Skin Disorder

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Published on : Nov 09, 2017

Stem cell research are making remarkable advances in the treatment of hard-to-cure and often life-threatening genetic diseases, significantly expanding the armamentarium of therapies available with the medical fraternity world over. Clinicians hoping to regenerate major parts of the human skin using genetically modified stem cells have experimented with transgenic stem cells of a patient suffering from epidermolysis bullosa.

The experimental gene therapy was chosen by the doctors at Katholisches Klinikum Bochum in October 2015, when none of the conservative or surgical therapies made any difference. The medical team used transgenic stem cells to cover defects and wounds spread across almost 80% of his entire body surface. The treatment bore fruit and the seven-year-old child boy, after being discharged in February, 2016, is reportedly living a normal, socially-active life and the reconstructed areas of the skin have since showed no complications.

The medical team at the Ruhr-University Bochum, Germany performed the extensive treatment consisting of a series of surgeries with the help of researchers at the Center for Regenerative Medicine at the University of Modena. The researchers believe this is the first time transgenic stem cells have been used to successfully reconstruct the entire human epidermis. The treatment is detailed in a report published in journal Nature.

Around 80% of Child’s Skin Transplanted Using Gene-Therapy Grafts

Epidermolysis bullosa is as a life-threatening congenital skin disease considered largely incurable is characterized by the formation of painful blisters and leads to open chronic wounds. According to an estimate, around 5, 00,000 people worldwide have been affected with the illness. The medical team obtained the stem cells from the unaffected parts of the child’s skin using biopsy and transferred the gene into the acquired stem cells using retroviral vectors to facilitate gene transfer.

The patient’s cells were notably affected by a gene mutation, which the researchers corrected by treating the cells with an engineered virus. They then grew a large number of these cells into sheets, which were then grafted onto the entire affected surface of the child’s body, following which intensive care was provided to help him recover.

In the years to come, the researchers believe that the breakthrough treatment will serve as a useful template for discovering stem cell-based therapies—ex vivo—for traumatic skin diseases affecting a large area of the body.