Targeted gene addition in human CD34 + hematopoietic cells for correction of X-linked chronic granulomatous disease

NATURE BIOTECHNOLOGY(2016)

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摘要
Targeted genetic correction of hematopoietic stem cells is applied to X-linked chronic granulomatous disease.
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关键词
Preclinical research,Genetic engineering,Genetic transduction,Stem-cell research,Genetic vectors
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