"A new way to manipulate embryonic stem cells (ESCs) offers hope for an eventual cell-based therapy to treat muscular dystrophies." All muscle cells have a similar embryonic origin. MD is caused by a loss of Dystrophin, a first team protein in muscle cells that loses the ability to do its job when a certain genetic mutation exists. An inability to grow new muscle is the result. Replacing defective cells with embryonic cells might solve the problem. But previous attempts to do this have resulted in too few new muscle cells being created within an acceptable time frame.
Turning the embryonic cells into muscle depends on a protein (one that binds certain segments of dna) called Pax3. The idea put forward by Rita Perlingeiro and her colleagues in the Perlingeiro Laboratory, Department of Developmental Biology, UT Southwestern Medical Center at Dallas suggests that if embryonic stem cells are cured in a petri dish and not exposed to the standard process of cell differentiation then Pax3 could be introduced directly through genetic manipulation.
This was accomplished with mice in the context of Duchenne muscular dystrophy. The new stem cells were delivered through the circulatory system rather than through direct injection into the muscles, resulting in an improvement in muscle function. Researchers accomplished this in a way that did not result in tumor formation. The intent now is to learn how to induce Pax3 action in embryonic stem cells without the need for genetic manipulation.
Read the Science Daily story.
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