Genetics

Genome editing helps decipher a congenital liver disease

Congenital hepatic fibrosis (CHF) is a rare genetic disease that causes malformation and fibrosis (scarring) of the liver. Occurring in roughly one out of every 20,000 births, CHF can lead to an enlarged liver, impaired blood ...

HIV & AIDS

Second HIV remission patient rekindles cure hope

For just the second time ever an HIV patient is in sustained remission from the virus in what was hailed by experts Tuesday as proof that the AIDS-causing condition could one day be curable.

Medical research

New method improves transplant safety in mice

Blood stem cell transplants—also known as bone-marrow transplants—can cure many blood, immune, autoimmune, and metabolic disorders, from leukemia to sickle-cell disease. But to make sure the healthy blood cells "take," ...

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Hematopoietic stem cell transplantation

Hematopoietic stem cell transplantation (HSCT) is the transplantation of blood stem cells derived from the bone marrow (in this case known as bone marrow transplantation) or blood. Stem cell transplantation is a medical procedure in the fields of hematology and oncology, most often performed for people with diseases of the blood, bone marrow, or certain types of cancer.

With the availability of the stem cell growth factors GM-CSF and G-CSF, most hematopoietic stem cell transplantation procedures are now performed using stem cells collected from the peripheral blood, rather than from the bone marrow. Collecting peripheral blood stem cells provides a bigger graft, does not require that the donor be subjected to general anesthesia to collect the graft, results in a shorter time to engraftment, and may provide for a lower long-term relapse rate.

Hematopoietic stem cell transplantation remains a risky procedure with many possible complications; it has traditionally been reserved for patients with life-threatening diseases. While occasionally used experimentally in nonmalignant and nonhematologic indications such as severe disabling auto-immune disease and cardiovascular disease, the risk of fatal complications appears too high to gain wider acceptance.

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