Uncovering a Hidden Risk: Gene Therapy's Role in Terry Horgan's Tragic Death
Originally Published 2 years ago — by STAT
A detailed investigation into the death of a 27-year-old man with Duchenne muscular dystrophy who received a CRISPR-based gene therapy treatment suggests that he likely died from an adverse effect of the virus used to deliver the therapy. The virus, known as adeno-associated virus (AAV), is generally considered benign but can cause side effects and, in rare cases, deaths at high doses. The patient experienced an acute respiratory distress syndrome (ARDS) triggered by an immune response to the virus. The study highlights concerns about giving high doses of AAV, particularly to older patients, and underscores the need for safer delivery methods in gene therapy.