Gene editing to cure sickle cell disease

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Sickle Cell Disease was the first molecular disease described in man, and is the most prevalent. In some African countries, India and the Middle East, up to 20% of the population carry the sickle gene mutation. In developing countries, 90% of children die before 5 years of age. In developed countries, patients suffer a lifetime of chronic pain and die ~20 years early. We will employ new gene editing approaches to repair the mutation or recruit fetal hemoglobin to cure SCD in human samples.