The quest for a viral victory
By Lindsay Chung

OTTAWA — All around the world, young boys are slowly wasting away from Duchenne Muscular Dystrophy. There is still no cure for this debilitating neuromuscular disease, and scientists have been working feverishly for years to come up with a way to combat the effects that leave most patients in a wheelchair before the age of 10.

Dr. Robin Parks has muscled his way into the arena with his innovative research into viral vectors. He studies the use of adenoviruses, spherical viruses containing double-stranded DNA, to deliver genes into animal models of genetic and acquired diseases, such as Duchenne Muscular Dystrophy (DMD) or cancer.

Parks is trying to reverse the effects of DMD, which affects mainly males. People with DMD are born without the dystrophin gene and, therefore, lack dystrophin protein. Dystrophin protein is vital for the normal functioning of cells required for muscle contraction and stretching. Without dystrophin, the patient’s muscles waste away.

Parks adds the dystrophin gene to the adenovirus and then injects the virus into a mouse. The virus binds to the outside of the cell and takes over, hopefully causing the cell to produce dystrophin protein.

While using adenovirus vectors for gene therapy is common, Parks’ research is unique because he uses helper-dependent adenoviruses, which have been stripped of all their viral genes. The immune system can tell when viral protein has entered the body, and it attacks and destroys the cell that has been infected with a virus, so Parks hopes helper-dependent adenoviruses will bypass this. So far, he says, these have proven to be much more successful than other adenoviruses in animal models of DMD.

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