Salk scientists adapt gene-editing system to correct dementia in lab

Salk Institute scientists reported that a form of dementia may eventually be treated with technology they have invented based on CRISPR technology.

The CRISPR/ Cas9 system makes it possible to rapidly and precisely alter DNA, the essential molecule of life. Salk Institute scientists reported that they've invented a new version of the technology that works on RNA — the messenger molecule that DNA relies on to carry out its instructions — and leaves DNA untouched.

Working in cultures of brain cells, the scientists corrected a protein imbalance that causes frontotemporal dementia with parkinsonism. More work needs to be done before it can be tried in patients, but they are preparing to test CasRx in animals, said Patrick Hsu, leader of the study published in the journal Cell.

CRISPR/Cas9 acts like molecular scissors on DNA. It has been likened to the impact of a word processor on writing. But any mistake could cause permanent damage to the genome, which has caused scientists to be cautious about developing it for therapy. But the new method's effects are not permanent because it targets RNA, shown at right, leaving the DNA intact, Hsu said.

Perhaps even more important is the novel method the scientists used to discover this new family of CRISPR, said Floyd Romesberg, a scientist at the Scripps Research Institute. Their approach could unlock a trove of new genetic engineering tools.