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Editorial
. 2022 Feb 4;6(2):e671.
doi: 10.1097/HS9.0000000000000671. eCollection 2022 Feb.

The EHA Research Roadmap: Hematopoietic Stem Cell Gene Therapy

Luigi Naldini  1   2 , Maria Pia Cicalese  1   3 , Maria Ester Bernardo  1   2   3 , Bernhard Gentner  1   4 , Michela Gabaldo  1   5 , Giuliana Ferrari  1   2 , Alessandro Aiuti  1
Affiliations
Editorial

The EHA Research Roadmap: Hematopoietic Stem Cell Gene Therapy

Luigi Naldini et al. Hemasphere. .
No abstract available

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Figures

Figure 1.
Figure 1.
Bone marrow-resident hematopoietic stem cells and hematopoietic progenitor cells replenish blood and tissues with new mature cells. Both hematopoietic stem cells and hematopoietic progenitor cells express the cell surface marker CD34, which is used to enrich a mixture of hematopoietic stem and progenitor cells for transplantation and gene therapy. Hematopoietic stem cells can be classed as long-term hematopoietic stem cells (LT-HSCs) or short-term hematopoietic stem cells (ST-HSCs). ST-HSCs progressively acquire lineage specifications to differentiate into lineage-committed progenitors and eventually terminally differentiated cells, which are released into the peripheral blood. A simplified scheme of human hematopoiesis is presented here. Alternative models have been postulated on the basis of cell surface marker analyses, in vitro and in vivo functional assays, clonal tracking by insertion analyses in hematopoietic stem and progenitor cell gene therapy studies, and single-cell RNA analyses (reviewed previously). Mendelian genetic disorders can affect self-renewal, differentiation, and/or the function of different blood and immune cells. Examples of genetic diseases for which gene therapy is under investigation or approved are represented in white boxes below affected cell types. Wiskott–Aldrich syndrome affects platelets and other lineages. CDP = common dendritic progenitor; CID = combined immunodeficiency; CLP = common lymphoid progenitor; CMP = common myeloid progenitor; GMP = granulomonocytic progenitor; LMMP = lymphoid-myeloid primed progenitor; MEP = megakaryocytic–erythroid progenitor; MPP = multipotent progenitor; NK cell = natural killer cell; preB = pre-B cell; preT = pre-T cell; SCID = severe combined immunodeficiency. Reprinted with permission from Nat Rev Genet. 2021;22:216–234.
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References

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