Current Research
Research involving triacetin supplementation has shown promise in a rat model. Triacetin, which can be enzymatically cleaved to form acetate, enters the brain more readily than the negatively charged acetate. The defective enzyme in Canavan disease, aspartoacylase, converts N-acetylaspartate into aspartate and acetate. Mutations in the gene for aspartoacylase prevent the breakdown of N-acetylaspartate, and reduce brain acetate availability during brain development. Acetate supplementation using Triacetin is meant to provide the missing acetate so that brain development can continue normally.
A team of researchers headed by Paola Leone are currently at the University of Medicine and Dentistry of New Jersey, in Stratford, New Jersey. The brain gene therapy is conducted at Cooper University Hospital. The procedure involves the insertion of six catheters into the brain that deliver a solution containing 600 billion to 900 billion engineered virus particles. The virus, a modified version of AAV, is designed to replace the aspartoacylase enzyme. Children treated with this procedure to date have shown marked improvements, including the growth of myelin with decreased levels of the n-acetyl-aspartate toxin.
Read more about this topic: Canavan Disease
Famous quotes containing the words current and/or research:
“I have come to believe ... that the stage may do more than teach, that much of our current moral instruction will not endure the test of being cast into a lifelike mold, and when presented in dramatic form will reveal itself as platitudinous and effete. That which may have sounded like righteous teaching when it was remote and wordy will be challenged afresh when it is obliged to simulate life itself.”
—Jane Addams (1860–1935)
“The great question that has never been answered and which I have not get been able to answer, despite my thirty years of research into the feminine soul, is “What does a women want?””
—Sigmund Freud (1856–1939)