Table 1 Engraftment of sorted haematopoietic cells in NSG.

	*SF3B1* mutation CD34 ⁺ cells	Mouse model	Cells injected	Engrafted mice	hCD33% cells hCD19% cells (12 weeks)	*SF3B1* MAB in CD34 ⁺ cells (day 0)	*SF3B1* MAB in HEC (12 weeks)
Lin⁻CD45⁺CD34⁺CD38⁻
HSC CD45RA⁻CD90⁺CD49f⁺	H662Q	NSG	1,629	1/1	100% 0.00%	46%	46%
MPP CD45RA⁻CD90⁻CD49f⁻	H662Q	NSG	29,944	0/1	NA	NA	NA
Lin⁻CD45⁺CD34⁺CD38
MLP CD45RA⁺CD90⁻	H662Q	NSG	51	0/1	NA	NA	NA
CMP CD45RA⁻CD135⁺	H662Q	NSG	16,231	0/1	NA	NA	NA
GMP CD45RA⁺CD135⁺	H662Q	NSG	28,217	0/1	NA	NA	NA

FACS, fluorescence-activated cell sorting; GMP, granulocyte macrophage progenitor; HEC, human engrafted CD45⁺CD33⁺ cell; HSC, haematopoietic stem cell; MAB, mutant allele burden; MDS, myelodysplastic syndrome; MDS-IC, MDS-initiating cell; MDS-RS, MDS patients with ring sideroblast; MPP, multipotent progenitor; NA, not applicable; NSG, NOD/SCID/IL2rγ^−OD mice.
The table shows the engraftment of FACS-isolated haematopoietic cell fractions injected into NSG mice. Progenitor cells from one MDS-RS patient were isolated and injected separately into NSG mice. After 12 weeks, mice were killed and the bone marrow was analysed. Only the rare HSC fraction gave rise to a myeloid-restricted engraftment, indicating the nature of the MDS-ICs in this rare HSC fraction.

Quick links

Search