World’s first patient treated with personalized CRISPR gene editing therapy through CHOP and Penn Med collaboration

A child diagnosed with a rare genetic disorder has been successfully treated with a customized CRISPR gene editing therapy by a team of researchers at Children’s Hospital of Philadelphia and Penn Medicine. The researchers targeted the infant’s specific variant of a gene that codes for an enzyme in the liver that converts ammonia to urea (which is later excreted in urine). The researchers designed and manufactured a gene-editing therapy delivered via lipid nanoparticles to the liver in order to correct the infant’s faulty enzyme. As of April, the infant had received three doses of the therapy with no serious side effects.