Gene Editing Saves Infant Girl’s Life

At Great Ormond Street Hospital (GOSH), the world’s first patient with “incurable” leukemia was cured today.  The patient, Layla Richards, was diagnosed with leukemia at the age of only 14 weeks old.  She went through months and months of treatment to cure it.  It eventually evolved into an incurable disease with no possible treatment.  The Richards family didn’t care what had to be done, they had to get their little girl better.  The researchers at GOSH said that they had been working on a treatment on genes in order to get rid of the aggressive leukemia.  The catch was that it has been only tested on mice before this moment.  The Richards, as stated before, didn’t care.  They wanted the treatment.  They volunteered their little girl.  They gave her the treatment which was an injection of 1ml of a genetically modified T-Cell called UCART19.  This is a T-Cell taken from a healthy donor and then genetically edited to be a “universal” cell that could be used for many different recipients.  Usually the cells would be rejected, but now they will be accepted.  The team did this by using molecular scissors called TALEN proteins in order to go into the “data” of the cell and cut off the gene in the T-Cell that would give away that it is a foreign cell.  The newly created gene was injected into Layla’s body and then continued to fight off the leukemia.  Several months later, One year old Layla has absolutely no more cancer cells left in her body.  Now the researchers are planning to do a widespread experiment in early 2016 to further the use of this miracle gene.

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