preview

Muscular Dystrophy Essay

Decent Essays

Duchenne Muscular Dystrophy and Ground-Breaking Research
Duchenne Muscular Dystrophy or DMD for short is a genetic disease that affects the skeletal muscles causing muscle degeneration and muscle wasting. Duchenne Muscular Dystrophy is an X-linked recessive chromosomal disease, which is caused by mutations in the DMD Gene (Regenerative Medicine). This Disease affects 1 in 3600 boys (Regenerative Medicine). A male born with this disease experiences respiratory dysfunction, trouble ambulation, cognitive impairment, some even experience premature death. Unfortunately, there is no cure for DMD, but there are research studies in the works for different therapies to help reverse this gene mutation and to elevate symptoms associate with this disease. …show more content…

The researchers are looking at a method of using this gene replacement in a way that a virus would infect a host. Meaning the scientist would engineer a virus that has healthy dystrophin genes within in it, then they will use this virus to infect the host, being someone who has DMD, in hopes that the engineered virus will act like a real virus and replicated once it has entered. Once the healthy genes have been replicated the muscles can uses these health dystrophin genes for muscle repair to reverse this disease. Getting into Myosin inhibitors, this therapy is used to stop Myostatin from being produced. Myostatin is a protein produced naturally by the body to stop muscle growth, In DMD patients muscle growth is detrimental, stopping this proteins production in would allow muscle to continue to grow in DMD patients. The Pharmaceutical Company Pfizer is currently developing a myostatin inhibitor which is currently undergoing clinical trials to be used I DM patients(MDA). In Stem cell Transplant in DMD patients, researchers are hoping to accelerate the rate of muscle repairer. Scientist are doing research with how stem cells can be used to repair damaged muscles and tissues in DMD patients. Stem cells play a huge role in repair and maintance in the body, taking healthy stem cells from a donor and transplanting them

Get Access