CRISPR Treatment Cuts Cholesterol by Up to 57% in a Single Shot

Scientists have improved upon a form of gene-editing therapy, creating an experimental treatment that looks to hold great promise for treating high cholesterol – a diagnosis affecting tens of millions of Americans, and linked to a number serious health complications. In new research conducted with mice, researchers used an injection of a newly-formulated lipid nanoparticle to deliver CRISPR-Cas9 genome editing components to living animals, with a single shot of the treatment reducing levels of low-density lipoprotein (LDL) cholesterol by up to 56.8 percent. In contrast, an existing FDA-approved lipid nanoparticle (or LNP; a tiny, biodegradable fat capsule) delivery system could only manage to reduce LDLs by 15.7 percent in testing. Of course, these results have so far only been demonstrated in mice, so the new therapy will take a lot of further testing before we know it’s both safe and equally effective in humans. But based on these results so far, signs are promising.

The way the treatment works relates to a gene in humans called Angiopoietin-like 3 (Angptl3), which produces proteins that inhibit the breakdown of certain fats in the bloodstream. People with a mutation in this gene tend to have lower amounts of fatty triglycerides and cholesterol in their blood – without showing other kinds of health complications – and for years scientists have been trying to recreate the process, with treatments that effectively mimic the effects of the mutation.

If we can replicate that condition by knocking out the Angptl3 gene in others, we have a good chance of having a safe and long term solution to high cholesterol,” says biomedical engineer Qiaobing Xu from Tufts University. “We just have to make sure we deliver the gene editing package specifically to the liver so as not to create unwanted side effects.

In the new research, Xu’s team developed a new formulation of LNPs called 306-O12B to target the gene, producing therapeutic effects in wild-type C57BL/6 mice that lasted at stable levels for 100 days after just a single injection of the treatment.

In addition to the cholesterol reduction, the experiment produced a 29.4 percent decrease in triglycerides in the animals’ blood, whereas the FDA-approved delivery method showed only a 16.3 percent reduction.

The findings are reported in PNAS.

Stem Cells Used To Create First Living Robots

Be warned. If the rise of the robots comes to pass, the apocalypse may be a more squelchy affair than science fiction writers have prepared us for. Researchers from the University of Vermont and Tufts University have created the first living machines by assembling cells from African clawed frogs into tiny robots that move around under their own steam.

One of the most successful creations has two stumpy legs that propel it along on its “chest”. Another has a hole in the middle that researchers turned into a pouch so it could shimmy around with miniature payloads.

CLICK ON THE IMAGE TO ENJOY THE VIDEO

These are entirely new lifeforms. They have never before existed on Earth,” said Michael Levin, the director of the Allen Discovery Center at Tufts University in Medford, Massachusetts. “They are living, programmable organisms.”

Roboticists tend to favour metal and plastic for their strength and durability, but Levin and his colleagues see benefits in making robots from biological tissues. When damaged, living robots can heal their wounds, and once their task is done they fall apart, just as natural organisms decay when they die.

Source: https://www.uvm.edu/
AND
https://www.theguardian.com/