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Redirecting N-acetylaspartate metabolism in the central nervous system normalizes myelination and rescues Canavan disease.
Gessler, Dominic J; Li, Danning; Xu, Hongxia; Su, Qin; Sanmiguel, Julio; Tuncer, Serafettin; Moore, Constance; King, Jean; Matalon, Reuben; Gao, Guangping.
Affiliation
  • Gessler DJ; Department of Microbiology and Physiological Systems.
  • Li D; Horae Gene Therapy Center, University of Massachusetts, Worcester, Massachusetts, USA.
  • Xu H; University Hospital Heidelberg, Centre for Child and Adolescent Medicine, Division of Child Neurology and Metabolic Medicine.
  • Su Q; Ruprecht-Karls University, Medical School, Heidelberg, Germany.
  • Sanmiguel J; Horae Gene Therapy Center, University of Massachusetts, Worcester, Massachusetts, USA.
  • Tuncer S; Horae Gene Therapy Center, University of Massachusetts, Worcester, Massachusetts, USA.
  • Moore C; University of Science and Technology of Kunming, China.
  • King J; Horae Gene Therapy Center, University of Massachusetts, Worcester, Massachusetts, USA.
  • Matalon R; Horae Gene Therapy Center, University of Massachusetts, Worcester, Massachusetts, USA.
  • Gao G; Heinrich Heine University, Medical School, Duesseldorf, Germany.
JCI Insight ; 2(3): e90807, 2017 02 09.
Article in En | MEDLINE | ID: mdl-28194442

Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Genetic Therapy / Canavan Disease / Amidohydrolases / Genetic Vectors Type of study: Prognostic_studies Limits: Animals / Humans Language: En Journal: JCI Insight Year: 2017 Document type: Article Country of publication:

Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Genetic Therapy / Canavan Disease / Amidohydrolases / Genetic Vectors Type of study: Prognostic_studies Limits: Animals / Humans Language: En Journal: JCI Insight Year: 2017 Document type: Article Country of publication: