Muscular Dystrophy is a set of hereditary diseases that cause muscles to deteriorate over time. The genes that carry the instructions for vital muscle proteins have undergone mutations, which is the cause of this. A gene-modulating therapy that momentarily restores the activity of these proteins can now be used to treat certain patients with these disorders. However, this approach is not always successful and occasionally provokes an unfavorable immunological reaction. It's not a treatment.

Unfortunately, many muscular dystrophies still have life-limiting effects despite recent breakthroughs in treatment. Researchers are constantly pushing the boundaries of biomedical science in an effort to improve the future for people with these illnesses.

From Modeling to Stem Cell Therapy

Skeletal muscle, or the muscles that are linked to bones, often has a remarkable capacity to recover from injury. The repair process depends heavily on muscle stem cells, especially a subtype known as satellite cells. Because of this, scientists have focused on satellite cells as a therapeutic potential approach for muscular dystrophies.

It is possible for the recipient to reject the donor stem cells, as is the case with all transplants. The dystrophin gene should be fixed using the patient's own cells, which have been taken and genetically altered. Nevertheless, genetic alteration can reduce the potency othe f stem cells.

Developing Stem Cells

Creating sufficient stem cells for treatment is another difficulty. Most of the body's muscles are impacted by numerous muscular dystrophies, including DMD, thus a lot of customized stem cells would be needed for treatment. After being obtained, stem cells must be multiplied (i.e., increased in number) in vitro before being given to the patient.

The ability of stem cells to produce new muscle tends to decline during in vitro growth. For stem cells to act both in vitro and in vivo, a very specialized environment is required, according to research. If this method of treatment and disease modeling is to be successful, it is imperative to comprehend how the environment impacts muscle stem cells.

Maintaining stem cells

Coating muscle stem cells with a material that resembles their natural environment are one method to aid the cells in maintaining their beneficial characteristics in vitro. This substance may also contain nanosystems substances that work in conjunction with biological systems—or different kinds of muscle cells.

Another strategy is to genuinely develop artificial muscles in vitro by combining extracellular matrix and other muscle cells. This kind of muscle regeneration can partially restore the functionality of injured muscles. It can be utilized as a helpful model for researching how the disease develops, just like stem cells can. These procedures have a great chance of healing injured muscles. However, it is still a way off until these methods can be used to treat diseases like DMD, which affects the entire big muscle group.

Challenges with Stem Cell Therapy

As it may promote the creation of new muscle fibers in injured muscles, this therapy has the potential to help persons with disorders that cause muscle wasting. Before stem cell therapy is accepted as a treatment for muscular dystrophy victims, researchers must overcome a number of obstacles. These comprise:

  • generating vast quantities of stem cells in the lab without sacrificing their regenerative abilities
  • identifying a good delivery method to ensure that the stem cells are delivered to every afflicted muscle
  • enhancing the engraftment of stem cells into the muscle to maximize their ability to generate new muscle cells
  • avoiding the implanted stem cells being rejected by the immune system.
  • The Future of Stem Cell Therapy for Muscular Dystrophy

    There is indeed a lot of active stem cell research for diseases that cause muscle loss, but it is unclear which kind of stem cell has the greatest therapeutic promise. Each type of stem cell being researched by scientists has advantages and disadvantages.

    Although satellite cells which occur naturally in the muscle might seem like an ideal candidate for stem cell therapy, they have a number of drawbacks. Satellite cells lose their ability to regenerate when growing outside of the body, which prevents them from forming a significant volume of muscle when transplanted. They also depend on the environment of the host muscle to work properly, therefore they might only be partially functional in a dystrophic muscle.

    In addition to serving as disease models, stem cells can also be used as a direct treatment for muscular dystrophy, which makes them an important tool in the investigation of the condition. Recent studies have suggested that stem cell treatment for muscular dystrophy may be able to stop the progression of muscle dystrophies by rejuvenating the cellular framework as well as restoring dystrophin function in patients. Exon-skipping, one of the newest genomic procedures, is providing new approaches for addressing the issues associated with the gene abnormalities that cause disorders like DMD.

Author's Bio: 

Advancells is a research-focused company in India that specialises in providing stem cell therapy for a variety of chronic and degenerative diseases. The company specializes in providing stem cells for preclinical testing, stem cell therapy, and even as in vitro research tools for clinical trials.