July 21, 2011
Gene Therapy In Rabbits Prevents Artery Clogging

Rabbits the world over are celebrating the good news that a gene therapy for rabbits prevents clogging up of arteries. Rats and mice seethe in jealousy and resentment.

A one-dose method for delivering gene therapy into an arterial wall effectively protects the artery from developing atherosclerosis despite ongoing high blood cholesterol. The promising results, published July 19 in the journal Molecular Therapy, came from research in rabbits.

The gene therapy turns on a protein thought to be involved in delivering the benefits of high HDL blood cholesterol.

The deployed gene produces a protein that is likely responsible for the beneficial effects of high-density lipoprotein, or HDL, commonly known as good cholesterol.

This substance is apolipoprotein A-1, or apoA-1. It pumps out harmful cholesterol from the scavenger-type cells that ingest fats and congregate in early atherosclerotic lesions.

ApoA-1 appears to remove cholesterol from the lesions and is capable of transporting it to the liver, where it can be excreted from the body.

Why take daily drugs for decades when you can reprogram the body to do something better?

"Localized one-time gene therapy might someday be an alternative or an important adjunct to systemic drugs such as statins that patients take for decades," Dichek said. "In gene-therapy trials for other diseases, one-time treatments have shown efficacy for at least nine years and will likely continue to be effective indefinitely. Because atherosclerosis is a life-long threat, gene therapy that protects blood vessels for a lifetime makes a lot of sense."

What I'd like to see: a gene therapy that is tied to activator and deactivator drugs. If this gene therapy is done and lasts for decades you could find yourself with a serious problem if a dangerous side effect turned up 5 or 10 years later. Better to have some way to limit the duration of the therapy.

Share |      Randall Parker, 2011 July 21 07:31 AM  Biotech Gene Therapy

bmack500 said at July 21, 2011 8:39 PM:

I'm thinking the FDA wouldn't allow this, should it prove to work in humans... not to be cynical, but it would interfere with the statin market...

fresno dan said at July 22, 2011 2:37 AM:

Damn big money rabbits - I'd like to see some research that benefits humans

B Dubya said at July 22, 2011 3:45 PM:

I'm 60, skinny as a rail, and at 51, had a coronary blockage of 95%, because of plaquing.
I hope the bunny rabbits share the remedy. Soon.

Side effects in 5 to 10 years? I'd take my chances on that one.

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