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Muscular dystrophy is an inherited disease characterized by progressive degeneration of skeletal muscles. Severe muscle degeneration cripples both children and adults and in some forms, takes their lives as a result of the failure of the respiratory muscles. Presently there is no cure.

What are the different types of muscular dystrophy, and whom do they affect?

There are several types of muscular dystrophies, some of which include Duchenne, Becker, and limb-girdle. Duchenne muscular dystrophy (DMD) is the most severe form; DMD cripples 15,000 North American children and kills two each day. Progressive degeneration destroys the children's ability to walk and to breathe. These children almost never reach adulthood. This disease affects only males since the gene is linked on the X chromosome. Females are only carriers and do not display the symptoms of DMD. In Becker muscular dystrophy (BMD) and Limb Girdle muscular dystrophy (LGMD), the progression of the disease occurs slower than Duchenne.

A myoblast is an immature muscle cell. It is the only cell type that has the unique property of natural cell fusion. Unlike stem cells, myoblasts are destined to become muscle cells--not anything else. On the left is a single myoblast, and on the right is a culture of myoblasts.

The process of obtaining pure, viable myoblasts in large quantities is the result of over 30 years of arduous research. The amplification process involves taking a 2.0 gram muscle biopsy from a donor's quadriceps and culturing the muscle tissue over several weeks. By the end of the process, we are able to obtain billions of myoblasts. The purity and viability is greater than 95% as determined by desmin stain.

The entire process starting from the biopsy to the final injection of the myoblasts is protected by numerous patents and trade secrets worldwide. Please take the time to review our patent estate by clicking "Patents" at the top.

5. Myoblast Transfer Therapy, or MTT, is the therapeutic process whereby the normal human genome is transferred to a genetically abnormal subject through the injection of cultured myoblasts. The evidence of genetic repair is the production of a protein called dystrophin which plays a key role in the structural integrity of the muscle cell membrane. Dystrophin is not produced at all in Duchenne patients. The reason is that these patients lack the genetic code for dystrophin. In addition, the muscle cells which have already degenerated and died are replaced by the newly injected myoblasts. Muscle staining reveals that the injected myoblasts survived within the patients' muscles even after six years. Therefore, Myoblast Transfer Therapy serves two important functions:

1. Repair genetically abnormal muscle cells.
2. Replenish lost, deteriorated cells

The idea of MTT was first conceived by Dr. Peter K. Law in 1975 while he was doing animal experimentation, and it was first published in 1978 while he was an Assistant Professor in Vanderbilt University. Subsequent publications have firmly established that myoblast transfer significantly improved the muscle genetics, structure, function, animal behavior and life spans of dystrophic mice. Click here to read more about Dr. Law.

On February 15, 1990, the first myoblast transfer as developed by Peter K. Law, Professor of Neurology at the University of Tennessee-Memphis, was performed at LeBohneur Children's Medical Center in Memphis, TN. Between 8 and 10 million myoblasts were injected into 9-year-old Sam Looper's extensor digitorum brevis, the muscle that controls the extension of the big toe. Three months later, Dr. Law and his co-investigators showed that the injected muscle demonstrated an improvement in muscle strength, cell appearance, and the presence of dystrophin, a protein missing in DMD. This is the world's first human gene therapy and the result was published in Lancet on July 14, 1990.

Heart Cell Therapy (HCT) describes the application of myoblast transfer therapy for treatment of heart muscle diseases. HCT aims to repopulate the dying heart with live muscle cells, increasing heart contractility, thus improving the quality of life and lengthening the lifespan of heart patients. Please read more about Cell Transplants International's developments to combat the No. 1 killer of human beings by following the link in the What's New section.

What is the difference between autologous and allogeneic myoblasts? What are the pros and cons?

Autologous refers to one's own cells whereas allogeneic refers to another individual's cells. Thus, an autologous myoblast transplant would be injecting one's own cultured myoblasts back into oneself, and an allogeneic myoblast transplant would involve injecting another person's cultured myoblasts into oneself. For a muscular dystrophy patient, an autologous transplant would not be appropriate since the cultured myoblasts would still be genetically abnormal. So it seems that an allogeneic transplant would be necessary in the case of a muscular dystrophy patient. The figure below illustrates the pros and cons of autologous and allogeneic myoblasts in the application of HCT. (Click on the figure for a larger image)

How are Cell Transplants International's myoblasts different from other organizations' myoblasts?

Cell Transplants International is able to produce myoblasts with greater than 95% purity and viability in the tens of billions. CTI owns the patents and trade secrets which protect all aspects of myoblast production and transplantation. No other organization has the knowledge or experience of myoblast production that CTI does. Whereas CTI regularly produces 50 billion myoblasts at 95% purity per MTT for a MD patient, other produce 500 million myoblasts at 35% to 65% purity. If you are considering another program, you may want to ask about the purity and viability tests that they conduct and how many cells are to be injected. Our patent estate widely covers the injection of myoblasts to treat muscle-related diseases, which include the skeletal muscle in muscular dystrophy and the cardiac muscle in heart diseases.