Gene correction in human long-term hematopoietic stem cells (LT-HSCs) could be an effective therapy for monogenic diseases of the blood and immune system. Here we describe an approach for X-linked sSevere cCombined iImmunodeficiency (SCID-X1) using targeted integration of a cDNA into the ...
), with expression in the bone marrow that is limited to long-term (LT)-HSCs in mice. Using a mouse single-colour tri-mCherry reporter driven by endogenous Hoxb5 regulation, we show that only the Hoxb5+ HSCs exhibit long-term reconstitution capacity after transplantation in primary transplant...
Long-term reconstituting haematopoietic stem cells (LT-HSCs) are used to treat blood disorders via stem cell transplantation. The very low abundance of LT-HSCs and their rapid differentiation during in vitro culture hinders their clinical utility. Previous developments using stromal feeder layers, defin...
Long-term hematopoietic stem cells (LT-HSCs) have the ability to self-renew and differentiate into all blood cell lineages. Understanding the genetic networks that regulate LT-HSC function in the adult bone marrow requires inducible gene targeting and bone marrow transplantations. In this chapter we...
Long-term hematopoietic stem cells (LT-HSCs) have the ability to self-renew and differentiate into all blood cell lineages. Understanding the genetic networks that regulate LT-HSC function in the adult bone marrow requires inducible gene targeting and bone marrow transplantations. In this chapter we...
Long-term reconstituting haematopoietic stem cells (LT-HSCs) are used to treat blood disorders via stem cell transplantation. The very low abundance of LT-HSCs and their rapid differentiation during in vitro culture hinders their clinical utility. Previo
Hematopoietic stem cells (HSCs) have long been detectable in transplantation assays but the isolation of a pure population of human HSCs with long-term repopulating activity (LT-HSCs) has been difficult because of the lack of appropriatecell surface markers.Notta et al. (2011)reported a cell iso...
Bone marrow failure in these USP10-KO mice was associated with remarkable reductions of long-term HSCs (LT-HSCs) in bone marrow and fetal liver. Such USP10-KO fetal liver exhibited enhanced apoptosis of hematopoietic stem/progenitor cells (HSPCs) including LT-HSCs but not of lineage-committed ...
Long-term HSCs (LT-HSC) and Nestinhigh/LEPR−ve Perivascular stem cells (PerSCs) reside close to the endosteum and the oxygenated arterioles that perfuse the endosteal niche, whereas short-term HSCs (ST-HSCs) and LEPR+ve/NestinlowPerSCs are in close proximity to the sinusoidal vessels ...
Here, we demonstrate that conditional inactivation of the Pbx1 proto-oncogene in the hematopoietic compartment results in a progressive loss of long-term hematopoietic stem cells (LT-HSCs) that is associated with concomitant reduction in their quiescence, leading to a defect in the maintenance of ...