Gene Therapy Pumps Up Monkeys

Several years ago, scientists created a myostatin "knockout" mouse. Put simply, a knockout mouse has had one of its genes deleted. Scientists use this strategy to study the effects of a single gene (if the gene is missing, we can see what effects its absence has on the animal). The myostatin gene in mice (and other animals) prevents muscle growth and instead promotes deposition of fat. This gene is thought to have been evolutionarily conserved in order to promote formation of energy stores (fat) and restrict calorie consumption (by muscle) in animals who don’t have a guaranteed source of food. Check out what happened when researchers knocked out the myostatin gene in mice:



The mouse blew up like body builders on steroids! Funny enough, the mouse was appropriately nicknamed "Mighty Mouse." Because the function of myostatin appears to be conserved across species, the researchers are hopeful that the findings will be beneficial in shaping treatments for muscle-wasting diseases, like muscular dystrophy, in humans. Via NPR this week:

Scientists are a step closer to finding a treatment for people with diseases like muscular dystrophy, thanks to some muscle-bound monkeys.

The monkeys grew bigger thigh muscles after receiving a type of gene therapy, according to a new study published in the journal Science Translational Medicine. Because monkeys are genetically similar to humans, the success means this sort of gene therapy is likely to work on people too. [...]

The success means the team is ready to move on to people. Researchers want to begin with patients who have a disease called inclusion body myositis, Mendell says. It leaves thigh muscles so weak people can't stand up.

"If everything works out we should be in a clinical trial by next summer, Mendell says.

On the downside, this will likely mean that in about 20 years most of us will look like the meatheads on the Jersey shore.