A 19-year-old from Kelowna, British Columbia, has made history by becoming the first person cured of a rare inherited immune disorder after doctors used prime gene editing to correct a single DNA error in his blood-forming cells. The breakthrough shows that precise gene editing inside a patient’s cells can restore immune defenses when no transplant donor exists.
The patient, Ty Sperle, faced chronic granulomatous disease as a child, which made him susceptible to infections and required daily antibiotics and antifungal medications. After months of treatment at B.C. Children’s Hospital in Vancouver, his immune system has normalized, with blood tests showing he can respond to infections like normal.
The gene editing method used is prime editing, a technique that rewrites DNA without cutting both strands. Doctors removed Sperle’s blood stem cells, corrected the DNA error, and infused them back into his body. This approach avoids the immune clashes that can follow donor transplants and requires careful hospital monitoring.
While years of follow-up are needed to assess the safety and durability of this approach, data from a Phase 1/2 program showed both treated patients regained immune function within one month. The study’s success highlights the potential for gene editing to treat rare genetic diseases, but more trials and expansion of access are necessary to make it a reality.
Source: https://www.earth.com/news/a-19-year-old-canadian-man-becomes-the-first-human-cured-through-prime-gene-editing