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Gene therapy for late infantile neuronal ceroid lipofuscinosis: neurosurgical considerations
OBJECT: The authors conducted a phase I study of late infantile neuronal ceroid lipofuscinosis using an adeno-associated virus serotype 2 (AAV2) vector containing the deficient CLN2 gene (AAV2(CU)hCLN2). The operative technique, radiographic changes, and surgical complications are presented. METHODS...
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| Autors principals: | , , , , , , , , , , |
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| Format: | Artigo |
| Idioma: | Inglês |
| Publicat: |
2010
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| Matèries: | |
| Accés en línia: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3763702/ https://ncbi.nlm.nih.gov/pubmed/20672930 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3171/2010.4.PEDS09507 |
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