Stem Cell Research and Muscular DystrophyMuscular dystrophy (MD) is not a single disease, but a collection of related conditions. These range from Duchenne/Becker muscular dystrophy to distal dystrophy, congenital muscular dystrophy, Emery-Dreifuss muscular dystrophy, and more. Each condition is unique, yet they all share common symptoms and outcomes. For all conditions, the Muscular dystrophy (MD) is not a single disease, but a collection of related conditions. These range from Duchenne/Becker muscular dystrophy to distal dystrophy, congenital muscular dystrophy, Emery-Dreifuss muscular dystrophy, and more. Each condition is unique, yet they all share common symptoms and outcomes. For all conditions, the Centers for Disease Control points out that men are more likely to be affected than women, and the condition generally presents during childhood, or during the early teen years. Upper arms and legs are the first to show signs of weakness, but all parts of the body can ultimately be affected, including the spine, the stomach, the chest muscles, the heart, and even the brain. There is no cure for muscular dystrophy, although stem cell research could provide relief for sufferers and their loved ones in the future.

What Is Muscular Dystrophy?

Muscular dystrophy, while a collection of different conditions and syndromes, can be summed up as a condition in which muscles progressively become weaker, and ultimately waste away. It is hereditary, and is caused by genetic mutations that are passed from family member to family member. All forms of muscular dystrophy progress, and many patients ultimately require a wheelchair for mobility. In severe cases, they may also require aids for breathing, and may need surgery, as well. Medications, surgery, therapy and physical aids are available to help alleviate symptoms, but there is no cure for any of the many muscular dystrophies. However, ongoing research into stem cells has shed light on how these cells could possibly play a role in treating muscular dystrophies, even hinting that a cure might be possible in the future.

What Causes Muscular Dystrophy?

It's important to understand that every form of muscular dystrophy is different. Each type is specific to a particular gene, in which a specific mutation has occurred. The gene involved is responsible for creating a protein necessary for protecting muscle tissues from damage. Because of the mutation, proteins are not produced in sufficient quantities, or the genes quickly become exhausted. To illustrate, consider the most common type of muscular dystrophy - Duchenne muscular dystrophy. In this condition, the protein dystrophin is not produced in enough quantities, or may be missing completely. This means that muscle tissues are easily damaged. To repair that damage, muscle stem cells called satellite cells create myoblast cells. These actually repair the tissue damage. The problem is that the damage is so extensive that satellite cells exhaust themselves creating myoblasts and eventually become depleted.

What Are Stem Cells?

Stem cells are progenitor cells. That is, they are proto cells that can ultimately transform into almost any other type of cell. They predate all other cells in the human body, and are the first type created within a fertilized embryo. From that point, they begin to differentiate themselves, forming various organs and tissue types. Eventually, the only stem cells remaining in the body are organ or tissue specific - liver stem cells, or brain stem cells, for instance. Stem cells are most numerous and least differentiated during embryonic development. However, science has discovered that significant quantities of young stem cells can be sourced from umbilical cord blood, as well as from umbilical cord tissue. This blood can be analyzed and then banked, and used for treatments for a very wide range of medical conditions. These cells are called allogeneic stem cells, which differ from the other type of stem cells used today, which are autologous stem cells. Allogeneic stem cells are the more effective by far, and require no genetic matching, as they do not trigger an immune response within the body. Autologous stem cells are less effective, and are the patient's own stem cells. They are usually harvested from fat, and less often from bone marrow. However, because they are the patient's own cells, they are old, less energetic, and have shortened telomeres, meaning far fewer divisions are possible.

How Might Stem Cells Benefit Patients with Muscular Dystrophy?

There are several potential treatments currently being researched to benefit patients with muscular dystrophy. Researchers are pursuing a number of different goals with stem cells, as well. One of those is to alleviate the underlying cause of muscular dystrophy in the first place - the lack of supporting protein necessary for protecting muscle tissue. Researchers are attempting to induce stem cells to transform into protein-producing cells, which would then be able to augment the defective genes within MD sufferers. These cells would be transplanted into the muscle tissue of those with MD, and would preserve and possibly even restore muscle function and strength. Another avenue of exploration is to create a line of cells in a laboratory with healthy dystrophin genes. These cells would then be transplanted into an MD patient and would essentially replace the defective genes. Of course, these cells would need to be manufactured in large quantities to benefit a patient, and different genes would need to be replicated to treat different types of muscular dystrophy. However, at this time, it is illegal to manipulate stem cells that will be transplanted into humans.

Challenges Facing Stem Cell Treatment for Muscular Dystrophy

There are a number of challenges creating roadblocks for stem cell researchers. One of those is the fact that autologous stem cells are often attacked by the host's immune system. Using allogeneic mesenchymal stem cells from umbilical cord blood eliminates this risk, as they do not trigger an immune response. Another challenge is simply inducing stem cells to develop into healthy, reliable producers of the specific protein needed to protect muscle tissue within a laboratory setting. Researchers are still in the early stages of this, and different inducements would be required to treat each of the myriad muscular dystrophies. There is also the fact that low engraftment is a problem with cell therapies, resulting in a relatively low success rate currently. A final challenge comes from the issue of evenly distributing stem cells throughout a patient's muscles. The entire body can be affected by MD; thus, transplantation needs to be even across all muscle groups. This possibly may be achieved with intravenous administration of umbilical cord mesenchymal cells. Such treatment is available but is experimental and investigational presently.

In the Future

While research is still ongoing, there is a great deal of promise. Currently, there is no FDA-approved stem cell treatment for muscular dystrophy, but that will change in a relatively short time as research progresses, and new techniques and technologies are developed. "Centers for Disease Control points out that men are more likely to be affected than women, and the condition generally presents during childhood, or during the early teen years. Upper arms and legs are the first to show signs of weakness, but all parts of the body can ultimately be affected, including the spine, the stomach, the chest muscles, the heart, and even the brain. There is no cure for muscular dystrophy, although stem cell research could provide relief for sufferers and their loved ones in the future.

What Is Muscular Dystrophy?

Muscular dystrophy, while a collection of different conditions and syndromes, can be summed up as a condition in which muscles progressively become weaker, and ultimately waste away. It is hereditary, and is caused by genetic mutations that are passed from family member to family member. All forms of muscular dystrophy progress, and many patients ultimately require a wheelchair for mobility. In severe cases, they may also require aids for breathing, and may need surgery, as well. Medications, surgery, therapy and physical aids are available to help alleviate symptoms, but there is no cure for any of the many muscular dystrophies. However, ongoing research into stem cells has shed light on how these cells could possibly play a role in treating muscular dystrophies, even hinting that a cure might be possible in the future.

What Causes Muscular Dystrophy?

It's important to understand that every form of muscular dystrophy is different. Each type is specific to a particular gene, in which a specific mutation has occurred. The gene involved is responsible for creating a protein necessary for protecting muscle tissues from damage. Because of the mutation, proteins are not produced in sufficient quantities, or the genes quickly become exhausted. To illustrate, consider the most common type of muscular dystrophy - Duchenne muscular dystrophy. In this condition, the protein dystrophin is not produced in enough quantities, or may be missing completely. This means that muscle tissues are easily damaged. To repair that damage, muscle stem cells called satellite cells create myoblast cells. These actually repair the tissue damage. The problem is that the damage is so extensive that satellite cells exhaust themselves creating myoblasts and eventually become depleted.

What Are Stem Cells?

Stem cells are progenitor cells. That is, they are proto cells that can ultimately transform into almost any other type of cell. They predate all other cells in the human body, and are the first type created within a fertilized embryo. From that point, they begin to differentiate themselves, forming various organs and tissue types. Eventually, the only stem cells remaining in the body are organ or tissue specific - liver stem cells, or brain stem cells, for instance. Stem cells are most numerous and least differentiated during embryonic development. However, science has discovered that significant quantities of young stem cells can be sourced from umbilical cord blood, as well as from umbilical cord tissue. This blood can be analyzed and then banked, and used for treatments for a very wide range of medical conditions. These cells are called allogeneic stem cells, which differ from the other type of stem cells used today, which are autologous stem cells. Allogeneic stem cells are the more effective by far, and require no genetic matching, as they do not trigger an immune response within the body. Autologous stem cells are less effective, and are the patient's own stem cells. They are usually harvested from fat, and less often from bone marrow. However, because they are the patient's own cells, they are old, less energetic, and have shortened telomeres, meaning far fewer divisions are possible.

How Might Stem Cells Benefit Patients with Muscular Dystrophy?

There are several potential treatments currently being researched to benefit patients with muscular dystrophy. Researchers are pursuing a number of different goals with stem cells, as well. One of those is to alleviate the underlying cause of muscular dystrophy in the first place - the lack of supporting protein necessary for protecting muscle tissue. Researchers are attempting to induce stem cells to transform into protein-producing cells, which would then be able to augment the defective genes within MD sufferers. These cells would be transplanted into the muscle tissue of those with MD, and would preserve and possibly even restore muscle function and strength. Another avenue of exploration is to create a line of cells in a laboratory with healthy dystrophin genes. These cells would then be transplanted into an MD patient and would essentially replace the defective genes. Of course, these cells would need to be manufactured in large quantities to benefit a patient, and different genes would need to be replicated to treat different types of muscular dystrophy. However, at this time, it is illegal to manipulate stem cells that will be transplanted into humans.

Challenges Facing Stem Cell Treatment for Muscular Dystrophy

There are a number of challenges creating roadblocks for stem cell researchers. One of those is the fact that autologous stem cells are often attacked by the host's immune system. Using allogeneic mesenchymal stem cells from umbilical cord blood eliminates this risk, as they do not trigger an immune response. Another challenge is simply inducing stem cells to develop into healthy, reliable producers of the specific protein needed to protect muscle tissue within a laboratory setting. Researchers are still in the early stages of this, and different inducements would be required to treat each of the myriad muscular dystrophies. There is also the fact that low engraftment is a problem with cell therapies, resulting in a relatively low success rate currently. A final challenge comes from the issue of evenly distributing stem cells throughout a patient's muscles. The entire body can be affected by MD; thus, transplantation needs to be even across all muscle groups. This possibly may be achieved with intravenous administration of umbilical cord mesenchymal cells. Such treatment is available but is experimental and investigational presently.

In the Future

While research is still ongoing, there is a great deal of promise. Currently, there is no FDA-approved stem cell treatment for muscular dystrophy, but that will change in a relatively short time as research progresses, and new techniques and technologies are developed.

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