Three-year-old Ipek Kuzu has an extremely rare genetic mutation that disrupts a protein needed for DNA repair, causing the loss of brain cells. Now she’s become only the second person in the world to receive a customized “antisense oligonucleotide” drug designed to compensate for the DNA mistake by allowing her cells to splice together a functional version of the protein. The drug took Boston-based pediatrician and geneticist Tim Yu only months to create, heralding a new era of individualized genomic medicine. But it cost $2 million to manufacture and test—leading to questions about how soon “hyper-personalized” treatments for rare genetic disorders can be made accessible and affordable. Journalist Erika Check Hayden got to know the Kuzu family, and in this episode she chronicles Ipek’s journey, with help from Ipek’s father Mehmet and Technology Review biomedicine editor Antonio Regalado.
