Vertex gene-editing therapy shows promise for younger children with blood disorders
Summary:
Vertex Pharmaceuticals reported positive trial results for its CRISPR-based gene therapy (Casgevy) in children aged 5–11 with sickle cell disease and transfusion-dependent beta thalassemia: treated children remained free of painful crises or transfusions for extended periods, supporting plans to expand regulatory approval and use in younger patients. The data were presented to regulators and at major conferences and could reshape pediatric treatment options for these inherited blood disorders.
The procedure, called ventriculo-coronary transcatheter outward navigation and re-entry, or VECTOR, creates a new route for blood flow that is a safe distance away from the aortic valve.
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Through the genome-wide data of 26 ancient human remains from northern and southern East Asia, spanning 9,500 to 300 years ago, including some extracted from the Qihedong skull, they carried out a series of scientific analyses that supported a southern China origin for proto-Austronesians.
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