The lone volunteer in a gene-editing study targeting a rare form of Duchenne muscular dystrophy most likely died after having a reaction to the virus that delivered the therapy in his body
The lone volunteer in a gene-editing study targeting a rare form of Duchenne muscular dystrophy likely died after having a reaction to the virus that delivered the therapy in his body, researchers concluded in an early study.
Terry Horgan, 27, of Montour Falls, New York, died last year during one of the first tests of a gene-editing treatment designed for one person. Some scientists wondered if the gene-editing tool CRISPR played a part in his death. The tool has transformed genetic research, sparked the development of dozens of experimental drugs, and won its inventors the Nobel Prize in 2020.
But researchers said the virus — one used to carry treatment into the body because it doesn’t usually make people sick — combined with his condition, triggered the problems that ultimately killed him.
Horgan appears to have had a more severe immune reaction « than others receiving similar or slightly higher doses” of the virus, the authors wrote in the study, which has not yet been peer-reviewed.
Horgan was enrolled in an early-stage safety trial approved by the Food and Drug Administration.
