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在线翻译:
szdaily -> Lifestyle -> 
Gene-engineered skin grafts save boy with rare disease
    2017-11-10  08:53    Shenzhen Daily

EUROPEAN researchers used genetic engineering to grow skin grafts that replaced 80 percent of the skin on a 7-year-old boy with a rare genetic disease.

Now he’s back at school, leading a normal life, the researchers reported Wednesday in the journal Nature.

“The amount of coverage that (the team) was able to achieve on this patient and the impact that this has had on the patient’s life is really incredible,” said Dr. Peter Marinkovich of Stanford University, who also uses skin grafts to treat similar patients.

“It shows the promise of what we are doing.”

The boy, who is not being identified, has a rare genetic condition called junctional epidermolysis bullosa. This particular type of epidermolysis bullosa (EB) is caused by defects in genes that control the development of a membrane in the epidermis — the top layer of skin.

Kids with EB, sometimes called “butterfly children” because their skin is as fragile as butterfly wings, develop painful blisters when their skin layers rub against one another with any movement. They are vulnerable to infections and skin cancer and often die before age 30. There is no cure.

“Since birth, the patient had developed blisters all over his body, particularly on his limbs, back and flanks,” the researchers wrote. His blistered skin had been attacked by two bacterial infections that destroyed most of it.

Skin grafts usually don’t work for these patients because of the genetic mutations controlling how the skin grows. The boy had been given skin grafts from his father, but they did not last.

However, some earlier experiments had shown it might be possible to take some skin from a patient, replace the faulty gene using gene therapy, grow the skin into sheets in the lab, and then graft it back.

But this approach had only been tried on two children and only replacing small patches of skin. The treatment itself could kill the boy.

The desperate parents asked to try it, anyway.

“His epidermis is currently stable and robust, and does not blister, itch, or require ointment or medications.”

The team at Children’s Hospital at Ruhr University in Bochum, Germany teamed up with specialists across Europe, led by Michele De Luca of the University of Modena and Reggio Emilia in Italy, a specialist in regenerative medicine.

They took a patch of non-blistered skin from the boy’s leg and used a virus to carry a corrected version of the bad DNA into his skin cells.

They grew grafts of the corrected skin and, in three separate operations over several months, replaced the missing skin.

The child was in terrible shape. He’d lost much of his body weight and was kept sedated at first because of the agonizing treatment.

But the grafts took hold, and grew, the team reported.

“He was discharged from the hospital in February 2016,” De Luca told reporters in a telephone briefing.

Tests showed that the gene therapy corrected immature cells known as stem cells. These stem cells replenished the skin cells with normal, healthy skin. (SD-Agencies)

 

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