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Stem Cells act as “Trojan Horse” to deliver gene therapy to brain, spinal cord
Posted on Tuesday, October 14, 2008 (EST)
Amyotrophic lateral sclerosis (ALS) researchers have shown that transplanted bone marrow stem cells can attach themselves to injured areas in the brain or spinal cord, possibly providing a way to deliver future gene therapy.
 
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Amyotrophic lateral sclerosis (ALS) researchers have shown that transplanted bone marrow stem cells can attach themselves to injured areas in the brain or spinal cord, possibly providing a way to deliver future gene therapy. Dr. Stanley H. Appel, chairman of neurology, The Methodist Hospital, Houston, Texas. Photo Credit: The Methodist Hospital, Houston, Texas

October 14, 2008, (Sawf News) - Amyotrophic lateral sclerosis (ALS) researchers at The Methodist Hospital in Houston have shown that transplanted bone marrow stem cells can attach themselves to injured areas in the brain or spinal cord, possibly providing a way to deliver future gene therapy.

The study found that transplanting bone marrow stem cells that are closely matched to the patients’ own bone marrow allowed for a significant percentage of those cells to travel to and reside in the brain or spinal cord. However, it is clear from the study that unless the bone marrow stem cells are engineered to secrete neuroprotective factors, such transplants are not likely to be beneficial in human ALS.

Appel, co-founder and co-director of the Methodist Neurological Institute at The Methodist Hospital and his research team have focused on bone marrow because of all stem cells, they offer a readily available source for potential use in the development of therapies, drug delivery and drug administration in ALS and other neurodegenerative diseases.

Commonly known as Lou Gehrig’s disease, ALS is a progressive neurodegenerative disease characterized by degeneration of the upper and lower motor neurons in the brain and spinal cord, which stimulate skeletal muscle movement. As more motor neurons die, muscle weakness becomes progressively worse. Sporadic (non-inherited) ALS affects 90 percent of the ALS population, and is representative of the patients at Methodist’s MDA/ALS Clinic, the first and one of the largest multi-disciplinary ALS clinics in the nation.

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