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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Hauptverfasser: Souweidane, Mark M., Fraser, Justin F., Arkin, Lisa M., Sondhi, Dolan, Hackett, Neil R., KaMinSky, Stephen M., Heier, Linda, Kosofsky, Barry E., Worgall, Stefan, Crystal, Ronald g., Kaplitt, Michael g.
Format: Artigo
Sprache:Inglês
Veröffentlicht: 2010
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Article
Online Zugang: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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