Stem cell advance could be key step toward treating deadly blood diseases

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Mirabai Vogt-James

Scientists at the UCLA Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research have made several discoveries that are critically important to understanding how blood stem cells are created and maintained — both in the body and in the laboratory.

The findings may lead to the creation of a new source of life-saving blood-forming stem cells, which could help people with a wide range of blood diseases by reducing many of the risks of bone marrow transplants. Thousands of people each year in the U.S. alone are diagnosed with diseases that could be treated with the procedure.

The study, led by senior author Dr. Hanna Mikkola and first co-authors Vincenzo Calvanese and Diana Dou, was published in the journal Nature Cell Biology.

Blood-forming stem cells, or hematopoietic stem cells, are found in the bone marrow and can create any type of blood cell. The researchers pinpointed the function of a cluster of specialized genes that play a key role in creating and preserving hematopoietic stem cells and identified the process by which those genes are activated during human development and in the laboratory.

For decades, doctors have used bone marrow transplants to treat people with diseases of the blood or immune system. One complicating factor has always been that certain proteins in the donor’s and recipient’s cells must match so that the donor’s immune system doesn’t reject the transplant. But finding a perfect match can be difficult, and the process is risky for both donors and recipients.

The UCLA research could provide a way to create patient-specific hematopoietic stem cells, which would reduce some of the challenges associated with bone marrow transplants.

“Our work focuses on finding a way to generate a supply of these life-saving hematopoietic stem cells in the lab so that they are a perfect match to the patient in need of a trans