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Clonal tracking in gene therapy patients reveals a diversity of human hematopoietic differentiation programs

10 Feb 2020

Abstract excerpt

In gene therapy with human hematopoietic stem and progenitor cells (HSPCs), each gene-corrected cell and its progeny are marked in a unique way by the integrating vector. This feature enables lineages to be tracked by sampling blood cells and using DNA sequencing to identify the vector integration sites. Here, we studied 5 cell lineages (granulocytes, monocytes, T cells, B cells, and natural killer cells) in...

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PMID: 32040546
DOI: 10.1182/blood.2019002350

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