Muscular Dystrophy (MD) is a genetic condition that causes the muscles in your body to weaken and degenerate over time. There are several types of Muscular Dystrophies that can occur, like Becker’s Muscular Dystrophy, Limb-Girdle Muscular Dystrophy, etc. Of these, Duchenne Muscular Dystrophy (DMD) is known to be the most fatal one. However, researchers are exploring novel techniques like stem cell treatment to alter the genetic mutations causing the illness and improve the patient’s quality of life.

What Happens in Duchenne Muscular Dystrophy (DMD)?

This condition occurs when there are mutations in the dystrophin gene that code specific muscle proteins. Dystrophin is responsible for the proper functioning of the muscles, and the lack of it can cause the muscle fibres to be in disarray and degenerate. As a result of improper functioning of the muscles, there is progressive and rapid muscle weakness and loss.

The Dystrophin gene found in the X-chromosome affects men, while most women only remain dormant carriers of the gene. Hence, the condition is typically passed on from either parent to the child. However, in some cases, this is due to a new, spontaneous genetic mutation that occurs during pregnancy.

What Are The Signs of DMD?

The muscle weakness caused by this condition starts manifesting during early childhood days. Delay in achieving developmental milestones like sitting, walking, and even talking are signs that the child could be suffering from DMD. The condition usually starts with a loss of strength and function in the pelvis and thigh region, followed by the arms. The child has trouble standing up as a toddler and loses mobility by the time they are teenagers. Increased convex curvature of the spine and change in gait are also noticeable signs of DMD. Even the heart and lung functions are affected due to the weakening of the muscles in the region, causing breathing and other issues for the child as they grow older. This leads to a respiratory or cardiac failure by the time the patient reaches their twenties.

Future of DMD Treatment With Stem Cells

Although there is no cure for DMD at present, the advancement in medicines, gene therapy, stem cell treatments, etc., are providing greater hope for patients to live longer and feel better.

Ongoing research indicates that mesenchymal stem cells (MSC) can help relieve the DMD symptoms by aiding muscle cell regeneration and repairing damaged muscle cells. Since umbilical cord stem cells are at an early development stage, they can take the form of any cell. Therefore, using Umbilical Cord Mesenchymal Stem Cells (UCMSCs) increases the muscle cells’ functionality and replenishes the stem pool.

In a study conducted on mice by a team of Harvard Stem Cell Institute researchers, the outcome looks promising. The researchers transplanted healthy stem cells into the muscles of mice with muscular dystrophy and observed that these new cells improved the damaged muscles’ functions and made them contract. They also noticed that the healthy stem cells replenished the diseased stem cell pool, which could repair any damage to the muscles during future injuries.

This study further demonstrates the potential of stem cell therapy in treating patients with muscular degeneration caused by DMD or other types of muscular dystrophy. The banking of stem cells can prove to be a blessing for your family as well as the field of medical science.

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Duchenne muscular dystrophy or DMD is a serious genetic disorder that affects hundreds of children every year. This disorder leads to conditions like atrophy and muscle weakness. There is no cure for the disease, however, supportive care can be offered to take care of their lungs and heart but these arrangements can only extend their lives till 27.

Now what causes Duchenne muscular dystrophy so dangerous for children? Kids, suffering from DMD cannot produce dystrophin, a protein that helps to keep away the muscles from breaking. The main reason is, these people have gone through a mutation in their gene in the X chromosome, which is responsible for providing the blueprint for dystrophin. The genetic mutation may lead to a more critical situation where most parts of the dystrophin get affected and a result the body stops producing the protein completely.

For some people with DMD, there are so many errors in the gene that the body cancels out the affected protein the moment it’s created. However, what’s more interesting to see is, the researchers have concentrated on the genes that are completely flawed and that interrupt in reading blueprint to produce dystrophin.

In order to address the criticalness of this condition, researches have conducted a study on a mice model. Multiple groups of researchers have harnessed the technique of applying gene editing enzyme complex CRISPR in order to modify the genes of mice with DMD. The technique allows cutting the errors in the genetic code, so that their effects can simply be avoided. After that the body starts to read the gene and then produce a shorter version of the protein that manages to protect the muscles from breaking down so easily, much like the process to treat patients with Becker muscular dystrophy.

The process involves loading the CRISPR complex into a virus and then injecting it to the mice fetus with DMD. The researchers have found that the mice began to create the shortened protein once the virus is injected into them. This helped the mice to resume the power of their muscles, which gradually led to a concrete treatment of their disease. So, the research was quite successful on the mice model.

The findings have generated a thin window of hope for the people, suffering with Duchenne muscular dystrophy mutation as well. But the process was easier for mice, and the researchers know that it’s not going to be the same easy game for humans. Moreover, no matter how foolproof the technique is proved for the mice, it may not deliver the same results on humans.

The scientists have established that the CRISPR can cut away additions to the gene but it will still take some time to show if it can address other kinds of mutations at all. Researchers are also not sure about the reactions of CRISPR complex in humans. Chances are that they might get rejected by the human immune system altogether. If it does not prove to be a 100% fix to the disorder, it means that the technique will not be able to treat all patients with Duchenne muscular dystrophy mutation.

However, having said all these, it’s also true that this research is the first ever experiment to have shown success, even if on a mice model. And researchers are hopeful that with more clinical trials, they might achieve the desired results soon.

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