What Gene Therapy can do to Reduce Bad Cholesterol

If you’re confused about what gene therapy can actually accomplish, you have only to look toward Baylor College of Medicine, where researchers are trying to cure diseases that cause high levels of bad cholesterol. The first problem was that they needed to experiment on mice and there are no good mouse models for high cholesterol that mimic the human condition.

Like labs around the world, the scientists at Baylor first had to make a mouse that actually replicates the disease. Using genetic engineering techniques, they were able to create a human-like lipid system within a mouse using human proteins.

Karl-Dimiter Bissig, an assistant professor at Baylor and senior author on a newly released study published in Nature Communications, says, “Within the model we created we were able to show the presence of certain proteins that are only found in humans, meaning the whole lipid machinery is present and can be used to study the disease within the model rather than just a cell structure created to resemble the machinery.”

Researchers grafted liver cells from a human with familial hypercholesterolemia, a disease of runaway bad cholesterol levels that shortens life span dramatically, onto mouse liver cells. The result was the same disease in the mice and a human-like serum profile.

Once the model was created, the scientists could go to work. Using an adenovirus-based gene therapy, they replaced the missing LDLR gene in the mice and serum levels of cholesterol returned to normal.

This is the first instance of inducing a human metabolic disease in an experimental animal model by hepatocyte transplant and then curing it with gene therapy.

To your health and wealth,

Stephen Petranek
for The Daily Reckoning

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