2. What are the different types of muscular dystrophy, and whom do they affect?
3. What is a myoblast?
4. How are myoblasts produced?
5. What is Myoblast Transfer Therapy (MTT)?
6. When was the first myoblast transfer therapy, and what were the results?
7. What is Heart Cell Therapy (HCT)?
8. What is the difference between autologous and allogeneic myoblasts? What are the pros and cons?
9. How are Cell Transplants International's myoblasts different from other organizations' myoblasts?
2.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.
3. 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.
4. 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.
6. 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.
7.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.
8. 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)
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