NTSAD provides family services, research, and education for
Tay-Sachs, GM-1, Sandhoff, Canavan and allied diseases.
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Stem cells differ from other kinds of cells in the body. All stem cells - regardless of their source - have three unique properties:
These unique properties could be used for cell-based therapies in which stem cells that are genetically altered to produce the missing enzyme are delivered to the brain or central nervous system.
Stem cell therapy has a lot of promise to cure but faces formidable challenges to develop safe and effective therapies. Some of the challenges include:
The embryonic stem cell controversy is becoming less of an issue as scientists have recently discovered how to manipulate adult cells into a stem cell state but more work is necessary to understand their therapeutic properties and potential.