Journal
STEM CELLS AND DEVELOPMENT
Volume 26, Issue 2, Pages 71-76Publisher
MARY ANN LIEBERT, INC
DOI: 10.1089/scd.2016.0230
Keywords
bone marrow; gene therapy; HSC
Funding
- French National Institute of Health and Medical Research (INSERM)
- European Research Council (ERC Regenerative Therapy) [269037]
- European Union FP7 grant (CELL-PID) [261387]
- European Union H2020 grant (SCIDNet) [666908]
- European Research Council (ERC) [269037] Funding Source: European Research Council (ERC)
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When considering inherited diseases that can be treated by gene transfer into hematopoietic stem cells (HSCs), there are only two in which the HSC and progenitor cell distribution inside the bone marrow and its micro-environment are exactly the same as in a healthy subject: metachromatic leukodystrophy (MLD) and adrenoleukodystrophy (ALD). In all other settings [X-linked severe combined immunodeficiency (X-SCID), adenosine deaminase deficiency, Wiskott-Aldrich syndrome, and beta-hemoglobinopathies], the bone marrow content of the different stem and precursor cells and the cells' relationship with the stroma have very specific characteristics. These peculiarities can influence the cells' harvesting and behavior in culture, and the postgraft uptake and further behavior of the gene-modified hematopoietic/precursor cells. In the present mini-review, we shall briefly summarize these characteristics and outline the possible consequences and challenges.
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