CRISPR sheds light on rare pediatric bone marrow failure syndrome
Chromosomes (blue) have protective end caps called telomeres that help them maintain stability. In this image, the telomeres (green) are abnormally short, which leads to DNA damage that accrues over time. Using the gene editing technology CRISPR, scientists at Washington University School of Medicine in St. Louis have shed light on a rare syndrome that causes children to lose the ability to manufacture vital blood cells. The syndrome, dyskeratosis congenita, is characterized by shortened telomeres. Credit: Marquet Minor Using the gene editing technology CRISPR , scientists have shed light on a rare, sometimes fatal syndrome that causes children to gradually lose the ability to manufacture vital blood cells. The research, at Washington University School of Medicine in St. Louis, suggests new lines of investigation into how to treat this condition -- dyskeratosis congenita -- which is characterized by shortened telomeres. Telomeres, the protective caps on the ends of c...