Researchers from the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center have combined the latest genome editing techniques with stem cells to produce red blood cells. This new study is the first of its kind to combine the two technologies to produce red blood cells.

The research, recently published the journal Cell Stem Cell, may lead to a simple and cost-effective way to produce large amounts of red blood cells. The technique could be particularly useful for patients with blood disorders who are unable to use the blood available in blood banks.

Scientists had previously used in vitro stem cells to produce red blood cells, but the cost for each blood unit was significant — between $8,000 and $15,000.

Lead researcher Dr. Vijay Sankaran worked with his team to identify a gene that contains specific variations in its sequence. The gene, known as SH2B3, can increase the production of red blood cells.

Dr. Sankaran explains the approach: “There’s a variation in SH2B3 found in about 40 percent of people that leads to modestly higher red blood cell counts, But if you look at people with really high red blood cell levels, they often have rare SH2B3 mutations.”

The study used the gene to enhance the production of red blood cells by stem cells. They used a technique called RNA interference (RNAi) to alter the SH2B3 gene in the stem cells.

The scientists targeted hematopoietic stem and progenitor cells (HSPCs) which were from adults and umbilical cord blood.  They found that stem cells which had been modified could produce between three and seven times the amount of red blood cells as normal stem cells. The red blood cells produced were identical to normal red blood cells.

Dr Sankaran says that the RNAi-modified cells could not be used directly in humans, however the red blood cells produced could be used in transplants. The research may lead to commercially viable red blood cell production in laboratories that could save thousands of lives.

Source: Researchers Use Gene Editing To Produce Red Blood Cells In Laboratory

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