[Comment] Sickle cell disease: tipping the balance of genomic research to catalyse discoveries in Africa

The completion of the Human Genome Project and the use of CRISPR/Cas9 for gene editing have begun to transform the diagnosis and management of disease. Sickle cell disease has been considered a perfect model for genomic research because it is a monogenic disease that is common and causes substantial morbidity and mortality but has no cure. The recent use of gene editing to modify disease severity1 and a case report of a patient with sickle cell disease who received successful treatment with gene therapy2 highlight the potential for translating genome-based knowledge into health benefits.

Source: LANCET - June 16, 2017 Category: General Medicine Authors: Julie Makani, Solomon F Ofori-Acquah, Furahini Tluway, Nicola Mulder, Ambroise Wonkam Tags: Comment Source Type: research