A latest technique is full of promise, however, it has been used just in mice. Researchers have used a gene-editing technique, CRISPR-Cas9 to improve faulty genes in adult mice with Duchenne muscular dystrophy. The technique resulted in improvements in mice muscle function.
Usually, muscular dystrophies are genetic diseases, whose symptoms include weakened muscle tissue. Duchenne muscular dystrophy (DMD) is defined as the weakened skeletal and cardiac muscle that is caused by the lack of a structural protein called dystrophin.
In Duchenne muscular dystrophy, mutations in the dystrophin gene can change the protein’s structure and function. Similar to glue, the protein helps anchor muscle cells to the extracellular matrix.
When there is no functioning dystrophin, muscle cells get easily damaged due to repeated contractions and relaxations. Eventually, these weakened muscle cells die, leading to the characteristic muscular deterioration linked to DMD.
With progress in the disease, individuals generally males get confined to a wheelchair and eventually develop heart failure because of weakening and dead cardiac muscle.
DMD can be caused by several thousand possible mutations. Most of them are deletions, missing DNA, which leads to the damaged protein. Contrary to this, a promising DMD treatment includes the rational deletion of the dystrophin gene sequence for the production of a so-called microdystrophin.
In mouse models, many microdystrophins have already been constructed and demonstrated to enhance muscle function. Besides this, microdystrophins have also been used in dog models successfully, DMD’s only huge mammalian model. But, in these studies, microdystrophins need to be delivered to the animal, using a virus which can merely deliver genes in a particular size.