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Liver tumor in gene therapy recipient raises concerns about virus widely used in treatment

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Jocelyn KaiserIt’s troubling news that gene therapy researchers have long anticipated: A hemophilia patient injected with a virus carrying a therapeutic gene in a clinical trial has developed a liver tumor.

The U.S. Food and Drug Administration (FDA) has halted the associated clinical trials, and uniQure, the Dutch firm behind the studies, is now investigating whether the virus itself caused the cancer.Gene therapy experts say that’s unlikely.

The patient had underlying conditions that predisposed him to liver cancer. Still, scientists say it’s crucial to rule out any role for adeno-associated virus (AAV), the viral delivery system, or vector, that is used in hundreds of other gene therapy trials. “Everyone will want to know what happened,”.

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COVID-19
United States
United States
Cases: 46 252 513
Deaths: 750 423
Recovered: 0
India
India
Cases: 34 321 025
Deaths: 459 652
Recovered: 0
Brazil
Brazil
Cases: 21 835 785
Deaths: 608 235
Recovered: 0
United Kingdom
United Kingdom
Cases: 9 215 683
Deaths: 141 607
Recovered: 0
Russia
Russia
Cases: 8 494 589
Deaths: 237 619
Recovered: 0
Detail statistic
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