Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is a big problem: size. The genes that are dysfunctional in muscular dystrophies are often extremely large, and current delivery methods can't courier such substantial genetic loads into the body. A new technology, dubbed "StitchR," surmounts this obstacle by delivering two halves of a gene separately; once in a cell, both DNA segments generate messenger RNAs (mRNAs) that join seamlessly together to restore expression of a protein that is missing or inactive in disease.
Published in the journal Science , StitchR—short for "stitch RNA"— restored expression of large therapeutic muscle proteins to normal levels in two different animal models of muscular dystrophy. StitchR enabled expression of the protein Dysferlin, which is lacking in individuals with limb girdle muscular dystrophy type 2B/R2, as well as the protein Dystrophin, which is absent in patients with Duchenne muscular dystrophy. Duchenne muscular dystrophy is the most common early onset form of muscular dystrophy; many boys become wheelchair bound in their teens and die in their twenties.
People with limb girdle muscular dystrophy experience weakness and wasting in the shoulder, hip, and thigh muscles, and often have difficulty standing, moving, and doing everyday tasks. "Gene therapy is a powerful tool for delivering a healthy gene copy back to a patient's cells to correct genetic diseases , b.
