Tuesday, July 9, 2013

Can Stem Cells Help muscular dystrophy

Can Stem Cells Help muscular dystrophy.  Because Duchenne’s muscular dystrophy is caused by the deficiency of one gene product – the protein dystrophin – scientists are hopeful that stem cells will someday be viable options for treating this disease. As things stand, the two main applications of stem cells for treating Duchenne’s are as vehicles for delivering gene therapies that replace or repair the defective DMD gene, and/or as cells that regenerate damaged muscle.

Scientists have identified different stem cells that are myogenic or able to form muscle. They can be found in skeletal muscle, bone marrow, blood, fat, and other tissues. Although figuring out which stem cells are ideal for treating Duchenne’s is no mean feat, there are certain qualities that make some stem cells better candidates than others.

Scientists must be able to grow and expand stem cells in the laboratory under conditions that maintain the ‘stemness’ of the cells – that is the ability to differentiate into muscle cells and self-renew to maintain the muscle-forming stem cell pool. Ideally, the stem cell of choice should be amenable to a system-wide delivery technique given that skeletal muscle throughout the body can be affected in Duchenne’s. Early experiments which transplanted muscle-forming cells by intramuscular injection were foiled because the spread of the cells was far too limited to be of any benefit. Since then, researchers have deduced that the circulatory system is a better delivery route because the transplanted cells can be shuttled throughout the body via the blood.

Of course, once stem cells are transplanted, they must be able to survive, grow and migrate to the site of damaged skeletal muscle. If at all possible, the stem cell graft should be able to do this without eliciting an immune response that would lead to graft rejection in the recipient. As well as being able to repair or rebuild muscle fibres, the best stem cell candidate should also be able to make dystrophin in order to maximize the overall affect of improving muscle function in individuals with Duchenne’s.
Below are a few of the different stem cell types that scientists are currently experimenting with to assess whether they meet the ideal criteria for treating this disease.

http://www.stemcellnetwork.ca/index.php?page=muscular-dystrophy

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