exa-cel
Vertex pharmaceuticals,CRISPR therapeutics AG
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impressive results, presented by professor franco locatelli, for gene therapy based on #crispr for #sicklecelldisease and #betathalassemia. 100% of scd patients are free from vaso-occlusive crisis. waiting for more long-term follow up results! #eha2022 #youngeha 
MD, PhD, hematologist at @hospitalHCFMUSP and @hosp_einstein, São Paulo, Brazil. Focused on #hemoglobinopathies and #genetherapy