Duchenne muscular dystrophy is a genetic disorder that affects only boys. It is characterized by a defect in a protein in muscle fibres called dystrophin. This causes a progressive weakening of the muscles.
Stem cells are produced in the bone marrow. They are immature cells that have the potential to self-generate themselves into many different cell types in the body. Autologus stem cell therapy is when the patients own stem cells are used to treat the condition. Bone marrow or peripheral blood stem cells are collected from the patient and then frozen and stored until they are needed. Alternatively, the stem cells originate from a donor, this is the allongenic approach.
The stem cells are then implanted into the patient where they travel to the muscles affected. They can then self-generate into new healthy muscle cells and form functional muscle fibres to replace the ones that have been damaged.