Managing Expectations for CRISPR Miracles
Parents of children with rare diseases are asking: How long until our CRISPR miracle?
A recent breakthrough in gene editing drugs has sparked hope for families of children with rare diseases. In May, a photo of "Baby KJ," the first infant treated with a personalized gene-editing drug, was circulated worldwide.
The drug fixed a single misspelled letter in his DNA that caused a severe, life-threatening liver disease. This achievement was touted as the "First Personalized CRISPR Gene Editing Therapy."
Gene therapy could "cure rare childhood diseases."
As the mother of a child with PURA syndrome, a rare neurodevelopment disorder, I saw firsthand how families of children with rare genetic diseases reacted to Baby KJ's story.
Author's Summary
Gene editing therapies offer hope for rare disease treatment.