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Blood stem cell transplants for metabolic disorders of the brain?

Bone marrow being extracted for a hematopoietic stem cell transplant
A patient’s bone marrow is extracted for a hematopoietic stem cell transplant, or HSCT. Once just a last-resort treatment for cancer, HSCTs are now used for a growing list of conditions, including certain metabolic disorders affecting the brain. (US Navy/Wikimedia Commons)

The history of hematopoietic stem cell transplant (HSCT) starts with severe cancers of the blood or immune system, like relapsed leukemias or lymphomas. Today, HSCTs are no longer solely the treatment of last resort for cancer but is used to treat a growing list of pediatric and adult conditions.

Most of these are cancers and blood disorders, but in recent years, a new frontier has opened up for HSCT: treatment of metabolic diseases, in particular, ones that affect the function of the brain.

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