One Man’s “Trash” is Another Man’s Cure for Familial Hypercholesterolemia

In the ultimate feat of recycling, scientists at UCLA and the Howard Hughes Medical Institute have re-purposed the LeXis gene as a cholesterol-lowering agent.
A recent press release issued by UCLA Health reported that this strand, formerly referred to as “junk DNA,” may actually suppress cholesterol levels as part of a treatment to reduce plaque in people with familial hypercholesterolemia (FH).

The LeXis gene was previously referred to as “junk” DNA because it was thought to have no real purpose. But the Human Genome Project proved otherwise. Now, more than a decade later, the activity of the gene is being explored and its implications could be huge for 1.3+ million people in the US with FH and the 1 in 250 people worldwide with this inherited disorder that leads to premature heart disease.

The initial test was on mice, but researchers are hopeful that the results will reduce the build-up of fat in liver cells as well as lower cholesterol and blockage in the arteries in human subjects.

Researchers tested a single injection of LeXis to see if it could slow the development of heart disease by giving mice either LeXis or a control gene, and feeding them a 15-week diet consisting of food high in sodium and cholesterol. (The mouse equivalent of fast-food hamburgers and french fries.) Researchers then measured the progression of heart disease.

It worked! LeXis lowered cholesterol and blockages in the arteries, and the treatment appeared to reduce the build-up of fat in liver cells.

In the next phase of the study, researchers intend to confirm the findings in larger animals and test the therapy in combination with currently available treatments. This is the first study to show that scientists may be able to treat a human disease with gene therapy. Taking out the trash never had so much promise…

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