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Dementia Work Earns Bioengineer Recognition

San Diego-based bioengineer Patrick Hsu has expanded gene editing to RNA, as he continues his work to discover breakthroughs for dementia

A bioengineer at the Salk Institute for Biological Studies who is redirecting gene-editing technology to target dementia has been named to MIT Technology Review’s annual list of Innovators Under 35.

Patrick Hsu has expanded CRISPR-Cas9 gene editing to RNA, which takes DNA instructions to cells. Up until recently manipulating DNA has been the focus of CRISPR.

Last year, he published a paper in the journal Cell showing that the approach corrected the protein imbalance in the cells of a dementia patient. Salk says the technology opens up new possibilities in brain therapies.

Hsu told MIT Technology Review that as a child he watched the onset of dementia in his grandfather.

“He would get into my bed in the middle of the night, disoriented, not knowing where he was,” he said. “It really made me think, how can I help?”

Any potential breakthroughs in dementia — however early — typically cause a big splash given the hordes of past drug development failures and the disease’s debilitating nature.

A New Job for CRISPR

Hsu, who heads a lab at Salk, got his doctorate degree at Harvard University. There he worked with Feng Zhang, one of the inventors of CRISPR.

But Hsu later realized tailoring CRISPR to RNA could yield a more flexible technique, according to MIT. He discovered a family of CRISPR enzymes targeting RNA.

At 27 years old, Hsu easily made the cutoff for the MIT list. It recognizes those offering a glimpse into the face of technology today as well as in the future.

(He was also named to Forbes’ 30 Under 30 list.)

“We look forward to many more health-changing innovations from him in the years to come,” said Salk President Rusty Gage, in a statement.

Hsu, who received his bachelor’s degree from the University of California, Berkeley, isn’t the only one repurposing CRISPR for RNA.

San Diego biotech Locana recently raised a $55 million Series A round to advance several preclinical programs that seek to address dysfunctional RNA processing, in an attempt to get at the root cause of genetic diseases.

Its technology was developed in the lab of Eugene Yeo, a professor at UC San Diego and Locana co-founder.

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