The crowning achievement of 2015 has been successfully used in order to reverse several complications related to Duchenne muscular dystrophy. CRISPR used to cure a mouse of muscular dystrophy, a rare neurological condition that impacts 1 in every 5000 infants.
The groundbreaking study was published on Thursday, in the journal Science, detailing how the CRISPR gene editing method was used in order to cure an adult mouse of Duchene muscular dystrophy.
CRISPR or clustered regularly-interspaced short palindromic repeats are strands of prokaryotic DNA, that contain short iterations of base gene sequences. The repetitions between the genes are followed by formations known as spacer DNA.
Space DND is the direct result to the host’s exposure to different forms of bacterial viruses or plasmids. CRISPR gene editing can be done by using the Tye II CRISPR system. One of this variants is the Cas9 system, that can be used in order to transfer edited genes back to the host, by using a plasmid of another non-pathogen virus.
According to the researchers working for the project, it would seem that this is the first time that the CRISPR/Cas9 method was employed in order to cure a mammal of a genetic disease. Further refined, the method can be used in order to target human genes.
Here is more on Duchene muscular dystrophy. DMD is considered to be a rare genetical disorder. The disease is linked to a recessive and defective X chromosome, which can cause an aggressive form of muscular dystrophy. This disease leads to muscle degeneration and often ends in a premature death.
DMD doesn’t occur spontaneously. Duchene muscular dystrophy is a neuromuscular degenerative disease that usually occurs before the age of 6. The first area affected by this disease are the muscles surrounding the hips, pelvic area, thighs, shoulder and calves.
Other symptoms begin to resurface in time such as walking disorders, fatigue, muscle contractures, muscle fiber difformities, skeletal deformities and losing the ability to walk.
Up until now, there was no cure for this disease. But thanks to a team of medical researchers we could be looking at a new cure for DMD and other genetical diseases. The doctors working for the project were able to use the CRISPR\Cas9 method in order to cure an adult mouse of muscular dystrophy.
DMD is often caused by an imbalance of dystrophin, a protein which contributes to the healthy formation of our muscles. By using this method, which employs the use of non-pathogen viruses, the team was able to correct the genetical aberration.
Thus, they have observed that the muscles found in the mouse’s leg area started to regenerate.