Adeno-associated virus-1 illustration; the use of AAVs resurrected the gene therapy field, but companies are now testing the limits of a 20-year-old technology (File photo, Shutterstock)

Af­ter 3 deaths rock the field, gene ther­a­py re­searchers con­tem­plate AAV's fu­ture

Nicole Paulk was scrolling through her phone in bed ear­ly one morn­ing in June when an email from a col­league jolt­ed her awake. It was an ar­ti­cle: Two pa­tients in an Au­dentes gene ther­a­py tri­al had died, grind­ing the study to a halt.

Paulk, who runs a gene ther­a­py lab at the Uni­ver­si­ty of Cal­i­for­nia, San Fran­cis­co, had planned to spend the day lis­ten­ing to talks at the Amer­i­can As­so­ci­a­tion for Can­cer Re­search an­nu­al meet­ing, which was tak­ing place that week. In­stead, she skipped the con­fer­ence, can­celed every work call on her cal­en­dar and be­gan phon­ing col­leagues across acad­e­mia and in­dus­try, try­ing to fig­ure out what hap­pened and why. All the while, a sin­gle name hung in the back of her head.

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