Inserting CRISPR machinery into human cells to fight Huntington’s and Lou Gehrig’s

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The gene-editing tool CRISPR is based on a natural defense system embedded in bacterial cells that recognizes and destroys invading viral DNA.

What if we could add that same attack mechanism to our own cells? A biotech startup, Locana, is trying to do just that by inserting the CRISPR machinery into human cells to equip the body to fight Huntington’s disease and amyotrophic lateral sclerosis, also known as Lou Gehrig’s disease.

To do it, Gene Yeo, the company’s cofounder and a professor of cellular and molecular medicine at the University of California, San Diego, School of Medicine, is repurposing CRISPR to go after a different target: RNA, the messenger molecule involved in transferring and decoding the genetic information stored in DNA.

In diseases like ALS, Huntington’s and some types of muscular dystrophy, RNA builds up and makes aberrant proteins that cause disease.

Normally, CRISPR uses a slicing protein called Cas9 that recognizes and chops up the desired DNA, eliminating a mutated gene. Yeo and his team modified Cas9 to leave DNA alone and instead bind to and cut problematic RNA.

Knocking down these RNAs is only temporary, though. RNA constantly regenerates, so its level in cells eventually rebounds back to normal after a few days to a week.

The GLP aggregated and excerpted this blog/article to reflect the diversity of news, opinion, and analysis. Read full, original post: Arming Bodies with CRISPR to Fight Huntington’s Disease and ALS