Alpharetroviral Vector-mediated Gene Therapy for X-CGD: Functional Correction and Lack of Aberrant Splicing

Comparative integrome analysis has revealed that the most neutral integration pattern among retroviruses is attributed to alpharetroviruses. We chose X-linked chronic granulomatous disease (X-CGD) as model to evaluate the potential of self-inactivating (SIN) alpharetroviral vectors for gene therapy...

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Bibliographic Details
Main Authors: Kaufmann, Kerstin B., Brendel, Christian, Suerth, Julia D., Mueller-Kuller, Uta, Chen-Wichmann, Linping, Schwäble, Joachim, Pahujani, Shweta, Kunkel, Hana, Schambach, Axel, Baum, Christopher, Grez, Manuel
Format: Artigo
Language:Inglês
Published: Nature Publishing Group 2013
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Original Article
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC3589149/
https://ncbi.nlm.nih.gov/pubmed/23207695
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2012.249
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