Aspartoacylase gene transfer to the mammalian central nervous system with therapeutic implications for Canavan disease.
Leone P, Janson CG, Bilaniuk L, Wang Z, Sorgi F, Huang L, Matalon R, Kaul R, Zeng Z, Freese A, McPhee SW, Mee E, During MJ.
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Ann Neurol. 2000 Jul;48(1):27-38. doi: 10.1002/1531-8249(200007)48:1<27::aid-ana6>3.0.co;2-6.
Ann Neurol. 2000.
PMID: 10894213
Clinical Trial.