Among the most promising advances in medicine recently has been developing the ability to “heal” misbehaving genes by replacing defective elements with healthy ones through “gene therapy.” A recent case offers some startling evidence of how effective this approach may come to be.
Doctors have replaced nearly all of one young patient’s epidermis with healthy skin.The unidentified Syrian boy suffers from epidermolysis bullosa, a painful, and fortunately rare, genetic disease that causes its victims’ skin to blister and ulcerate outside or inside the body, depending on the form of the disease. People with this disease — typically appearing in infancy or early childhood — are at heightened risk of infections and sepsis, and tend to die young from skin cancer. The form of the disease the boy has is junctional epidermolysis bullosa, and it’s on the outer layer of his skin.
Junctional epidermolysis bullosa is caused by mutations in three genes — LAMA3, LAMB3 or LAMC2 — so gene therapy could offer a chance of getting this terrible condition under control. There has, though, been skepticism regarding the use of gene therapy for major skin lesions. So the results obtained by doctors Michele De Luca and Reggio Emilia and just published inNature are surprising to some, and unquestionably promising.
The gene therapy began in 2015 when the boy was 7. He’d been admitted…
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