Jun 1, 2023

Researchers link death in gene-editing study to a virus used to deliver the treatment, not CRISPR

Posted by in categories: biotech/medical, genetics

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.

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