Gene Therapy Stabilizes Tay-Sachs Disease Babies

Two patients at UMass Memorial Medical Center with Tay-Sachs disease received gene therapy to attempt to correct the mutation in the HEXA gene that causes the disease.  The patients that were treated have shown no negative effects and their disease has appeared to stabilize.  Tay-Sacs disease is a fatal genetic disorder in which nerve cells die and patients lose the ability to move or breathe.

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