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CMCC 2797

Novel and highly efficient production of red blood cells

Inventors: Vijay Sankaran

Invention Types: Therapeutics, Research Tool

Research Areas: Oncology/Hematology, Regenerative Medicine

Keywords: Stem/Progenitor Cell

For More Information Contact:  Meyer, Abbie

 

Invention Description:

Transfusion of red blood cells (RBCs) is an extremely common medical procedure used for a variety of lifesaving applications. However, the supply of RBCs is completely dependent upon the frequency and quality of blood donations, and blood banks are often in short supply. The ability to produce RBCs ex vivo from hematopoietic stem and progenitor cells (HSPCs) or pluripotent stem cells is a major area of research focused on providing a more reliable alternative to our volatile blood supply.


The lab of Vijay Sankaran at Boston Children’s has developed a new method to boost the efficiency of ex vivo production of RBCs. By silencing the gene SH2B3, the researchers were able to boost RBC production from adult HSPCs and cord blood HSPCs by 2- to 7- fold. In addition, deleting SH2B3 in human pluripotent stem cells increased the yield of erythroid cells by 3-fold.


To achieve this result, the researchers used large-scale population genetics to identify that rare loss-of-function versions of the SH2B3 gene are linked to increased RBC counts in humans. The lab used two independent gene silencing techniques, shRNA and CRISPR/Cas9 genome editing, and found that knockdown of SH2B3 led to increased differentiation of HSPCs into mature RBCs ex vivo. These findings demonstrate the principle that stable knockdown of SH2B3 can provide an improved method for generating RBCs.

Applications:

• Ex vivo method to produce RBCs from a population of stem or progenitor cells for use in blood transfusions or for the treatment of various disorders characterized by a deficiency of RBCs.

• Research approach that uses population genetics to identify rare gene variants linked to altered levels of specific cell types in order to develop new ways to boost production of specific cells for use in regenerative medicine.

Competitive Advantages:

• Inhibition or disruption of SH2B3 function improves upon current ex vivo methods for RBC production by:

o Increasing the quality and quantity of RBCs

o Decreasing the number of HSPCs initially required to produce RBCs, by an estimated one-fifth

o Decreasing the cost of RBC production

Business Opportunity:

Licensing and collaboration opportunities available

Key Publications: Felix C. Giani, Claudia Fiorini, Aoi Wakabayashi, Leif S. Ludwig, Rany M. Salem, Chintan D. Jobaliya, Stephanie N. Regan, Jacob C. Ulirsch, Ge Liang, Orna Steinberg-Shemer, Michael H. Guo, T├Ánu Esko, Wei Tong, Carlo Brugnara, Joel N. Hirschhorn, Mitchell J. Weiss, Leonard I. Zon, Stella T. Chou, Deborah L. French, Kiran Musunuru, Vijay G. Sankaran. Targeted Application of Human Genetic Variation Can Improve Red Blood Cell Production from Stem Cells. Cell Stem Cell. In Press. Online 22 October 2015

IPStatus: Pat. Pend.