CRISPR sheds light on rare pediatric bone marrow failure syndrome

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 suggests new lines of investigation into how to treat this condition — dyskeratosis congenita — which is characterized by shortened telomeres. Short telomeres lead to progressive DNA damage that accumulates over time.

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