?Gene therapy: Aka "aspartoacylase deficiency"or "aminoacylase deficiency" is a degenerative disorder of the nerve cells in the brain. It is an autosomal recessive disorder (when both parents are carriers, 25% chance of having an affected child). There is no cure-lithium citrate has been tried to decrease the level of n-acetyl aspartate. Gene therapy is in an experimental phase.
Answered 4/5/2015
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