Pathophysiology and Treatment of Canavan Disease.

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第一作者： David,Pleasure

第一单位： Institute for Pediatric Regenerative Research, Shriners Hospitals for Children Northern California and UC Davis School of Medicine, 2425 Stockton Blvd, 95817, Sacramento, CA, USA. depleasure@ucdavis.edu.;, C/o Shriners Hospital, 2425 Stockton Blvd, Sacramento, CA, 95817, USA. depleasure@ucdavis.edu.

作者： David,Pleasure ^[1] ; Fuzheng,Guo ^[2] ; Olga,Chechneva ^[2] ; Peter,Bannerman ^[2] ; Jennifer,McDonough ^[3] ; Travis,Burns ^[2] ; Yan,Wang ^[2] ; Vanessa,Hull ^[2]

作者单位： Institute for Pediatric Regenerative Research, Shriners Hospitals for Children Northern California and UC Davis School of Medicine, 2425 Stockton Blvd, 95817, Sacramento, CA, USA. depleasure@ucdavis.edu.;, C/o Shriners Hospital, 2425 Stockton Blvd, Sacramento, CA, 95817, USA. depleasure@ucdavis.edu. ^[1] Institute for Pediatric Regenerative Research, Shriners Hospitals for Children Northern California and UC Davis School of Medicine, 2425 Stockton Blvd, 95817, Sacramento, CA, USA. ^[2] Department of Biological Sciences, School of Biomedical Sciences, Kent State University, Kent, OH, 44242, USA. ^[3]

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