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CRISPR study reduces cholesterol in mice without "editing" DNA
Rather than hacking away with genetic scissors, recent work has instead used CRISPR to temporarily turn certain genes on and off through epigenetic modulation. In that vein, the new study developed a CRISPR-Cas9 repressor system that can silence a gene called Pcsk9, which regulates cholesterol levels. The Duke researchers managed to package this system and deliver it into the livers of adult mice.
"We previously used these same types of tools to turn genes on and off in cultured cells, and we wanted to see if we could also deliver them to animal models with an approach that is relevant for gene therapy," says Charles Gersbach, lead researcher on the study. "We wanted to change the genes in a way that would have a therapeutic outcome, and Pcsk9 is a useful proof-of-concept given its role regulating cholesterol levels, which in turn affect health issues like heart disease."
The researchers started with a Cas9 enzyme taken from the bacteria species Staphylococcus aureus, and to keep it from making cuts to the target DNA, they created a "dead" version dubbed dCas9. This was bundled with a KRAB protein that silences gene expression, and the combination was then packaged inside adeno-associated viral (AAV) vectors – viruses that are engineered to carry the active ingredients to the right DNA target.
In their tests, the Duke researchers delivered the system to adult mice, where it activated in their livers. Compared to a control group that had simple saline injections, the Pcsk9 genes in the test mice were successfully repressed and the animals' cholesterol levels dropped as a result. Better yet, the effects of a single treatment lasted six months.
RNA-guided transcriptional silencing in vivo with S. aureus CRISPR-Cas9 repressors (open, DOI: 10.1038/s41467-018-04048-4) (DX)
Related: CRISPR Used to Reduce Tissue Inflammation and Damage
CRISPR Used to Epigenetically Treat Diseases in Mice
(Score: 2) by realDonaldTrump on Tuesday May 01 2018, @06:00AM (1 child)
Some of these articles, it's like English is not their first language. Hard to understand. But I think they're saying it works on the bad cholesterol, the LDL. My bad cholesterol is a little high. It's not high. But it's almost high. I don't know why, I don’t think anybody knows. My doctors put me on some pills. But my bad cholesterol keeps going up. It's not a problem. But if it keeps going up, maybe, probably, it could become a problem. Dr. Jackson says I could live to 200 years old. Amazing genes. But maybe I could go even longer with the artificial ones.
Very disappointed at what the Dems did to Dr. Jackson. I never drink, I've never touched alcohol. But if a guy can drink a lot and still do good work, that's fine. And Dr. Jackson does good work. And he would have done great work for our vets, for our VA. He's a great guy, abused by a bunch of politicians that aren't thinking nicely about our country. Not nicely at all. I'll find another guy. Or a lady. Someone great, believe me. Need to privatize!!
(Score: 3, Interesting) by arslan on Tuesday May 01 2018, @11:47PM
I LDL really the source of bad stuff happening in the body though? Wasn't a theory out somewhere that high LDL is a reaction to something else bad happening in the body and supressing LDL, though beneficial for the direct symptoms caused by it is just turning a blind eye to the source which bad symptoms will still manifest eventually in the body?
Note, INAMD.