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Improving Gene Therapy Efficiency through the Enrichment of Human Hematopoietic Stem Cells

27 Jun 2017

Abstract excerpt

Lentiviral vector (LV)-based hematopoietic stem cell (HSC) gene therapy is becoming a promising clinical strategy for the treatment of genetic blood diseases. However, the current approach of modifying 1 × 10 8 to 1 × 10 9 CD34 + cells per patient requires large amounts of LV, which is expensive and technically challenging to produce at clinical scale. Modification of bulk CD34 + cells uses LV inefficiently,...

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PMID: 28663101
DOI: 10.1016/j.ymthe.2017.05.023

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