Two Encouraging Results with Stem-Cell and Gene Therapy

An Italian team has successfully treated Duchenne muscular dystrophy in dogs. Previous research by the team had shown that a special type of stem cell — a mesoangioblast stem cell — that is harvested from small blood vessels, and easily grown in tissue culture, can efficiently enter into and repair muscle. Using a retroviral vector, the researchers inserted partial copies of the dystrophin gene into mesoangioblast stem cells from the diseased animals. This intervention caused dystrophin production in muscle throughout the body and greatly improved normal muscle morphology and function.

A second Italian study involved a human patient with epidermolysis bullosa, a potentially fatal skin disease caused by a defect in the laminin 5 (LAM5) gene. …