Genetic treatments for sickle cell

May 01, 2016

sickle cell disease blood cells

Patients with sickle cell disease with high levels of fetal hemoglobin have a milder course of the disease because the mutation that causes red blood cells to “sickle” resides only in adult hemoglobin. Now researchers at Dana-Farber/Boston Children's Cancer and Blood Disorders Center and other centers are looking to gene editing or gene therapy – and eventually a pill – to control the switch to ease the devastating disease, Scientific American reports.

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Irene Sege
Senior Media Relations Specialist
irene.sege@childrens.harvard.edu
1-617-919-7379

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