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Article

Alpharetroviral vector-mediated gene therapy for X-CGD: functional correction and lack of aberrant splicing.

Molecular therapy : the journal of the American Society of Gene Therapy - 1 Mar 2013

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

Abstract excerpt

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 of monogenic diseases. Therefore, we combined the alpharetroviral vector backbone with the elongation factor-1α...

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Identifiers

PMID: 23207695
DOI: 10.1038/mt.2012.249
PMCID: PMC3589149

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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