A scientific adventure whose ingredients include the looming threat of a fatal disease, the decision to reinvent themselves as biologists, and the goal of silencing prions.
The clinical trial with antisense oligonucleotides, born of their efforts, is considered one of the most interesting trials of 2025, but this is only a part of the story. This married couple is also pursuing other avenues to halt the onset of prion diseases. In the summer of 2024, they published a study in Science using epigenetic editing in mice. Then, in January 2025, their experiments with base editing were published in Nature Medicine. Yet Sonia Vallabh was a newly graduated jurist, and her husband, Eric Minikel, was working in urban planning, when they discovered that she carried a mutation that would condemn her to die of fatal familial insomnia within two or three decades.
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It’s another CRISPR first: fixing a hereditary disease in utero. Scientists from Pennsylvania University and the Children’s Hospital of Philadelphia used a base editor to rescue tyrosinemia in fetal mice. “The results of this proof-of-concept work demonstrate the possibility of efficiently performing gene editing before birth, pointing to a potential new therapeutic approach for selected congenital disorders,” Avery C. Rossidis and colleagues write in 
CRISPeR FRENZY 