Gene Editing Improves Yield of Stem Cell-derived Red Blood Cells

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The need for red blood cells for medical transfusions is a significant need that cannot be overstated. The cost of obtaining sufficient blood necessary for transfusions is often prohibitively high, and the difficulty in sourcing blood has, as a result, an unfortunate human cost.

Stem cells have, as a result, appeared as a way to generate RBCs for medical purposes where sourcing ready blood is not possible.

However, so far, the yield of red blood cells (RBCs) produced from stem cells in vitro has not shown to be practical for routine transfusion needs. The issues come down to scaling up red blood cell production to quantities useful for clinical trials, which has proven to be a significant challenge.

Finding ways in which red blood cell production from stem cells can be improved is an area of growing research interest. Now, a new study claims to have identified, via genetic engineering, a new way to significantly improve the yield of RBCs derived from stem cells.

The study, titled “Targeted Application of Human Genetic Variation Can Improve Red Blood Cell Production from Stem Cells,was published in Cell Stem Cell by Dr. Vijay Sankaran’s lab at the Dana-Farber Cancer Institute in collaboration with colleagues at Boston Children’s Hospital and the University of Pennsylvania.

The authors identified that silencing gene SH2B3 – which traditionally encodes a negative regulator of cytokine signaling – leads to an increase in RBC generation. By suppressing SH2B3 in primary human CD34+ hematopoietic stem and progenitor cells with SH2B3-targeting shRNAs, the authors demonstrated that the yield of in-vitro-derived RBCs three- to five-fold. In addition to that, this caused differentiation of stem cells to occur significantly more rapidly.

 

Targeted Application of Human Genetic Variation Can Improve Red Blood Cell Production from Stem Cells

 

What this means practically, is that this technique could potentially improve RBC production at 80% less cost than traditional costs required to obtain equivalent amounts of RBCs.

The potential medical benefits are multi-fold, so it will be interesting to see further developments of this technology and whether further studies can support larger scale expansion.

We are closely monitoring this field, having recently published a manuscript to develop novel methodologies and cryopreservation solutions to freeze RBCs in ways that are safer for transfusion. Contact us to enquire.

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