Sonia Vallabh always thought genome editing wouldn’t be an option. It was such an obvious solution that people invariably asked, “What about CRISPR?” at the end of talks. But the details never made sense.
Vallabh and her husband, Eric Minikel, scientists at the Broad Institute, have been racing to develop a treatment for prion disease, a rare form of neurodegeneration that killed her mother at age 51 and would, if left untreated, likely kill Vallabh as well. It’s an insidious disease. Although it can have several causes, in her case, a single genetic misspelling will lead neurons to produce misfolded versions of otherwise workaday proteins called prions. These mutant prions jump cell to cell, like viruses, misfolding other prions they touch and forming toxic, neuron-killing chains.
So why not use CRISPR to repair or remove the mutant gene?
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