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DNA

Twenty-three years ago, the field of gene therapy was bursting with the promise of breakthrough treatments. Then it was almost instantly derailed by the death of an 18-year-old clinical trial volunteer named Jesse Gelsinger after he received a genetically engineered virus that had been developed to treat his rare liver condition.

An FDA investigation revealed that the principal investigator and/or the team running the gene therapy trial at the University of Pennsylvania failed to disclose that before Gelsinger was treated, other patients had experienced alarming side effects and that monkeys administered the same engineered virus had died. The incident, coupled with dangerous outcomes involving other gene therapy treatments, had a chilling effect on the field and investors backed away.

Today, new approaches to gene therapy that include advances driven by CRISPR gene editing tools are raising hopes of a gene therapy revival. There are potential breakthroughs in the pipeline, including treatments for different types of cancer and sickle cell disease.

I’m concerned that gene therapy 2.0 is at risk of making the same mistakes that plagued the 1.0... see more