Uncoupling N‑acetylaspartate from brain pathology: implications for Canavan disease gene therapy

11:31 am 20 Dec 2017

Available online: 1 November 2017


We thank K.-A. Nave for providing the CNP-Cre mice, and Matthias Eckhardt for the Nat8l cDNA. Nadine Mersmann helped generating foxed ASPA mice. The authors thank the Biological Resources Imaging Laboratory (MWAC, UNSW Sydney) for providing access to instrumentation and operational support, including MRI imaging with National Imaging Facility support. This study utilized the Australian Phenomics Network Histopathology and Organ Pathology Service, University of Melbourne.

N-Acetylaspartate, Canavan disease, Myelination, White matter disorder, Brain metabolism, Neurophysiology, AAV Gene therapy

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