Dual-regulated Lentiviral Vector for Gene Therapy of X-linked Chronic Granulomatosis

Regulated transgene expression may improve the safety and efficacy of hematopoietic stem cell (HSC) gene therapy. Clinical trials for X-linked chronic granulomatous disease (X-CGD) employing gammaretroviral vectors were limited by insertional oncogenesis or lack of persistent engraftment. Our novel...

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Detaylı Bibliyografya
Yayımlandı:Mol Ther
Asıl Yazarlar: Chiriaco, Maria, Farinelli, Giada, Capo, Valentina, Zonari, Erika, Scaramuzza, Samantha, Di Matteo, Gigliola, Sergi, Lucia Sergi, Migliavacca, Maddalena, Hernandez, Raisa Jofra, Bombelli, Ferdinando, Giorda, Ezio, Kajaste-Rudnitski, Anna, Trono, Didier, Grez, Manuel, Rossi, Paolo, Finocchi, Andrea, Naldini, Luigi, Gentner, Bernhard, Aiuti, Alessandro
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: Nature Publishing Group 2014
Konular:
Original Article
Online Erişim:https://ncbi.nlm.nih.gov/pmc/articles/PMC4435596/
https://ncbi.nlm.nih.gov/pubmed/24869932
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2014.87
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