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Hope it Ends Better than the Book

Daniel Keyes; classic science fiction story (and later novel) Flowers for Algernon tells of a mentally disabled man who is suddenly made a great genius via a surgical procedure. Before the procedure is performed on him, it is proved on a mouse -- named Algernon.

And now we have this:

In a case of life imitating art, researchers at the Massachusetts Institute of Technology (M.I.T.) reported today that they had successfully reversed mental retardation in mice... Now M.I.T. scientists report in Proceedings of the National Academy of the Sciences USA that they ameliorated brain damage in mice caused by a genetic disorder known as fragile X syndrome by blocking an enzyme involved in cellular development.

Fragile X affects one in 4,000 boys and one in 6,000 girls. It is caused by a mutation in the fragile x mental retardation 1 gene (FMR1)—located on the X sex chromosome— that results in the loss of the fragile x mental retardation protein (FMRP). The resulting illness is characterized by hyperactivity, attention deficit, repetitive behavior, anxiety and cognitive difficulties ranging from learning disability to mental retardation.

When studying the formation of dendrites for a 2004 paper, Mansuo Hayashi, a research affiliate in M.I.T.'s Picower Institute for Learning and Memory, discovered that these structures could be strengthened and altered to transmit information more efficiently by inhibiting nerve cell production of the enzyme called p21-activated kinase (PAK). PAK regulates actin, another protein, which shapes parts of the cell (including the dendrites). When PAK is inhibited, more actin is manufactured and the dendrites are able to properly mature.

What made Keyes' story a tragedy is the eventual reversal of the condition of both the man and the mouse subected to intelligence-enhacing procedure. While it's not clear what applicability this current research will have for human beings -- although it is bound to have some -- there wouldn't appear to be much risk that gene therapy will suddenly reverse itself.

So we'll stay tuned.


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