exa-cel
Vertex pharmaceuticals,CRISPR therapeutics AG
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oral breaking #eha2022 crispr #geneediting exa-cel leads to #transfusion independence in 95% patients in thalasssemia with normalisation hb level and reduction of blood need in all. contributed to this work. 
Haematologist, red cell disorders and marrow failure. Working towards definitive cure: #BMT #genetherapy. @ImperialNHS @ImperialImmuno @TheEBMT @ApothecariesLDN