Pathophysiology and Treatment of Canavan Disease

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This study was supported by Shriners Hospitals Grant 439043; NIH 1R21NS096004-01; and the Dana Foundation.

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Correspondence to David Pleasure.

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Special issue: In honor of Vittorio Gallo.

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Pleasure, D., Guo, F., Chechneva, O. et al. Pathophysiology and Treatment of Canavan Disease. Neurochem Res 45, 561–565 (2020).

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  • Canavan disease
  • Aspartoacylase (encoded by ASPA)
  • N-acetyltransferase 8-like (encoded by Nat8l)
  • NaDC3 (encoded by Slc13a3)
  • Vacuolar (“spongiform”) leukodystrophy
  • N-acetyl-l-aspartate (NAA)
  • Gene therapy