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Onset of gene editing in humans

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05.09.2018

CRISPS Therapeutics and Vertex Pharmaceuticals are the first companies to conduct a human trial of the gene editing technology.The trial will be carried out in Germany, while the gene therapy itself will undergo testing in patients with beta-thalassemia.

Vertex has already performed the first CRISPR-based treatment. The company acquired shares in CRISPR Therapeutics in exchange for the right to license as many as up to six programs for gene editing.CTX001 product will be developed to target sickle cell disease and beta-thalassemia.

Both disorders are caused by mutations within the beta-globin gene.CTX001 will use CRISPR gene editing ex vivo, namely "outside the body".Cells of given patients will be collected and edited in order to increase the level of fetal hemoglobin in their blood cells.Furthermore, such altered cells will be infused back to the original patient where they will produce blood cells with fetal hemoglobin, thus compensating for the previously defective adult hemoglobin.

Source: fiercebiotech.com

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