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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Detaylı Bibliyografya
Asıl Yazarlar: 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.
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: 2010
Konular:
Article
Online Erişim: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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