Team hits milestone for prion disease treatment. For them, it's personal.

Patient-scientist, husband among researchers who developed promising gene-editing therapy for rare, fatal condition.

New research provides hope that prion disease — a handful of rare, invariably fatal disorders caused by misfolded proteins in the brain — may, in the not-too-distant future, have a treatment if not a cure.

The work, published early this year in the journal Nature Medicine, showed that altering a single base in the gene that produces the killer proteins can reduce by half the amount of that protein in the brains of laboratory mice, a step that extended their lifespans 52 percent.

Authors of the work, at the Broad Institute of MIT and Harvard, caution that several potentially lengthy steps remain before human trials of the technique can be undertaken. Still, they agreed that the results indicate the pathway that they embarked upon nine years ago toward effective treatment in humans appears promising.

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