Doctors Give Boy New Skin, Gene Therapy Saves Life

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Doctors Give Boy New Skin, Gene Therapy Saves Life

DOCTORS SAVE BOY’S LIFE, DESIGN NEW SKIN

Last week, Italian researchers made a stunning announcement.  They essentially saved a seven-year-old boy’s life by giving him new skin.  The boy suffered from a brutal skin condition called JEB and faced a painful death.  But doctors were able to almost completely reconstruct all of the boy’s skin to save his life.  They used gene therapy to do it.  Their work and success is unprecedented.  These doctors give new hope where there was none before.

GENE THERAPY OFFERS TREATMENT TO UNTREATABLE

Gene therapy and stem cells are in the news a lot.  But they have often been cautionary stories about regulation or issues to make most people afraid.  But this breakthrough tells us that hopeless conditions may now be reconsidered as treatable.  The young boy and his affliction with JEB is a perfect example.

BOY GIVEN NEW SKIN FOR 80% OF BODY

JEB isn’t always life threatening.  But it’s a genetic disorder that leaves patients skin prone to infections and cancers.  Usually treatment of JEB is to treat local issues as they happen, with great care.  But this boy had a life-threatening case of JEB and needed a new skin for 80% of his entire body.  So researchers had to create a new treatment to save his life.  And that’s exactly what they did.

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ITALIAN DOCTORS GIVE HOPE WITH NEW MIRACLE TREATMENT

Last week the journal Nature published reports detailing how the researchers from Italy’s University of Modena and Reggio Emilia used gene therapy to save the boy’s life.  They took skin cells from the boy’s skin that had no blistering from JEB.  They cultured those cells and genetically modified them, correcting the gene that caused the disease.  But they then grafted the genetically corrected cells back onto the little boy.  After 21 months, more than 80% of the boy’s new skin had been regenerated with the corrected gene.  When doctors give new designed skin to patients, the future is now.

BOY’S LIFE SAVED AND ALMOST NORMAL, BUT FOR HOW LONG?

In the past, researchers had only been able to reconstruct a small patch on a patient’s skin.  But now we can hope that diseases like JEB can be effectively cured with treatment, not just managed by doctors.  But of course, we are talking genetics.  And in this case, a genetic disease.  We will have to wait and see if the little boy’s JEB comes back, or if a new cancer occurs from his modified skin.  It’s a new frontier to pioneer.  But for now, this little boy is alive and able to have a much more normal life, thanks to gene therapy and some medical wizards in Italy.

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