New Hope for Treating a Rare Genetic Disease Before Birth
Stanford Medicine scientists are opening a clinical trial for prenatal treatment of a rare blood disorder called Fanconi anemia. The study will test stem cell transplants performed before birth, using healthy blood-forming stem cells donated by the mother.
Evidence from preclinical studies in animals suggests that these transplants may be able to treat Fanconi anemia without any of the chemotherapy, radiation or immune suppression that are needed for typical stem cell transplants.
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