New Hope for Treating a Rare Genetic Disease Before Birth

Stanford Medication scientists are opening a medical trial for prenatal therapy of a uncommon blood dysfunction known as Fanconi anemia. The examine will take a look at stem cell transplants carried out earlier than start, utilizing wholesome blood-forming stem cells donated by the mom.

Proof from preclinical research in animals means that these transplants might be able to deal with Fanconi anemia with none of the chemotherapy, radiation or immune suppression which might be wanted for typical stem cell transplants.

“The analysis offers us actual cause to consider prenatal transplants would stabilize the bone marrow in these sufferers and forestall the necessity for subsequent therapies,” mentioned Stanford Medication pediatric hematologist Agnieszka Czechowicz, MD, PhD, who’s main the brand new examine.

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