When clinicians at the Children’s Hospital of Philadelphia (CHOP) treated baby KJ – born with a rare, often-fatal urea-cycle disorder – with a CRISPR...
Gene therapy for ultra-rare neurodegenerative diseases is no longer hypothetical, yet access remains uncertain and uneven. The case of August and his...
For people with advanced melanoma skin cancer, a treatment utilizing immune cells derived from their own tumours holds great promise. Özcan Met,...
Personalized gene-editing treatments remain out of reach for most patients in Europe today. To understand why, it helps to look at the front lines. At...
