Aspartoacylase gene transfer to the mammalian central nervous system with therapeutic implications for Canavan disease
- Author
- LEONE, P1 2 ; JANSON, C. G1 2 ; MCPHEE, S. W6 ; MEE, E7 ; DURING, M. J1 2 ; BILIANUK, L3 ; WANG, Z3 ; SORGI, F4 ; HUANG, L4 ; MATALON, R5 ; KAUL, R5 ; ZENG, Z2 ; FREESE, A1
[1] Department of Neurosurgery, CNS Gene Therapy Center, Thomas Jefferson University, Philadelphia, PA, United States
[2] Department of Neurosurgery, Laboratory of Neurogenetics, Yale University, New Haven, CT, United States
[3] Department of Radiology, Children's Hospital of Philadelphia, Philadelphia, PA, United States
[4] Department of Pharmacology, University of Pittsburgh, Pittsburgh, PA, United States
[5] Division of Genetics, University of Texas, Galveston, TX, United States
[6] Department of Molecular Medicine, University of Auckland, Faculty of Medicine, Auckland, New Zealand
[7] Auckland Hospital, Auckland, New Zealand - Source
Annals of neurology. 2000, Vol 48, Num 1, pp 27-38 ; ref : 55 ref
- Publisher
- Willey-Liss, Hoboken
- Publication country
- United Kingdom
- Document type
- Article
- Language
- English
- Keyword (fr)
- Animal Aspartoacylase Canavan maladie Enfant Essai clinique Etude cas In vivo Lipide Thérapie génique Traitement Transfert génétique Encéphale pathologie Enzyme Enzymopathie Homme Hydrolases Lipoïdose Métabolisme pathologie Sphingolipidose Système nerveux central pathologie Système nerveux pathologie
- Keyword (en)
- Animal Aspartoacylase Canavan disease Child Clinical trial Case study In vivo Lipids Gene therapy Treatment Genetic transfer Cerebral disorder Enzyme Enzymopathy Human Hydrolases Lipoidosis Metabolic diseases Sphingolipidosis Central nervous system disease Nervous system diseases
- Keyword (es)
- Animal Aspartoacylase Enfermedad canaván Niño Ensayo clínico Estudio caso In vivo Lípido Terapia génica Tratamiento Transferencia genética Encéfalo patología Enzima Enzimopatía Hombre Hydrolases Lipoidosis Metabolismo patología Esfingolipidosis Sistema nervosio central patología Sistema nervioso patología
- Classification
- Pascal
- 002 Biological and medical sciences / 002B Medical sciences / 002B22 Metabolic diseases / 002B22D Errors of metabolism / 002B22D02 Lipids (lysosomal enzyme disorders, storage diseases)
- Discipline
- Metabolic diseases
- Origin
- Inist-CNRS
- Database
- PASCAL
- INIST identifier
- 1428263
Sauf mention contraire ci-dessus, le contenu de cette notice bibliographique peut être utilisé dans le cadre d’une licence CC BY 4.0 Inist-CNRS / Unless otherwise stated above, the content of this bibliographic record may be used under a CC BY 4.0 licence by Inist-CNRS / A menos que se haya señalado antes, el contenido de este registro bibliográfico puede ser utilizado al amparo de una licencia CC BY 4.0 Inist-CNRS
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DOI
10.1002/1531-8249(200007)48:1<27::AID-ANA6>3.0.CO;2-6 
https://dx.doi.org/10.1002/1531-8249(200007)48:1<27::AID-ANA6>3.0.CO;2-6
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