Temple Street Children's University Hospital

healthcare IE

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1,646

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CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia

Haydar Frangoul , David Altshuler , Maria Domenica Cappellini +23 more

Transfusion-dependent β-thalassemia (TDT) and sickle cell disease (SCD) are severe monogenic diseases with severe and potentially life-threatening manifestations. BCL11A is a tr...

2020 New England Journal of Medicine 1646 citations

Institution Info

Type: healthcare
Country: IE
Publications: 1
Citations: 1,646

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ROR: https://ror.org/0527gjc91