Effective gene therapy for an inherited CNS disease in a large animal model

Autor:	John H. Wolfe, Mark E. Haskins, Charles H. Vite, Sumit N. Niogi, Kenneth J. Drobatz, Marco A. Passini, Joseph C. McGowan
Rok vydání:	2005
Předmět:	Pathology medicine.medical_specialty Time Factors Genetic enhancement Alpha-mannosidosis Genetic Vectors Central nervous system Neuropathology Biology Brain mapping Injections Viral vector Animals Genetically Modified Transduction Genetic alpha-Mannosidase medicine Animals RNA Messenger In Situ Hybridization Neurologic Examination Brain Mapping CATS Staining and Labeling Glycogen Storage Disease Type II Brain Genetic Therapy Human brain medicine.disease Magnetic Resonance Imaging Disease Models Animal Treatment Outcome medicine.anatomical_structure Neurology Cats Neurology (clinical)
Zdroj:	Annals of Neurology. 57:355-364
ISSN:	1531-8249 0364-5134
DOI:	10.1002/ana.20392
Popis:	Genetic diseases affecting the brain typically have widespread lesions that require global correction. Lysosomal storage diseases are good candidates for central nervous system gene therapy, because active enzyme from genetically corrected cells can be secreted and taken up by surrounding diseased cells, and only small amounts of enzyme (
Databáze:	OpenAIRE
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