Current research into treatments and a cure for DMD are mainly focussed on the areas of genetics, cellular chemistry and pharmacology.
Gene therapy
This area of research is focused on inserting the correct genetic code for the dystrophin protein into muscle cells. This will assist the cell with a “recipe” enabling it to produce dystrophin, and, hopefully this will lead to a cure.
Stem cells
Scientists are currently looking into the use of stem cells to encourage muscle cells to develop new muscle without the dodgy gene for DMD. There are currently great ethical debates about this form of research.
Pharmacological research
This form of research aims to develop better treatment options for DMD. The focus here is not necessarily on finding a cure but treating the symptoms of the disease thereby enhancing quality of life.
Replacement proteins
Scientists are currently working on proteins that can act on substitutes for dystrophin in the muscle cell. They are also currently looking into certain enzymes that clean up damaged muscle cells.
Figure 9: courtesy of science sisters
