Long-term follow-up of the first in human post-transcriptional genetic silencing of BCL11A in sickle cell disease in a phase 1 pilot and feasibility trial

Research
Written By Christine Briscoe

Authors: Erica Esrick, Leslie Lehmann, Amy Federico, Heather Daley, Colleen Dansereau, Satiro DeOliveira, John Everett, Pei-Chi Kao, Boya Liu, Theodore Moore, Emily Morris, Helene Negre, Kit Shaw, Gavin Roach, Jerome Ritz, Aoife Doto, Olivia Silva, Helene Vincon, Frederic Bushman, Donald Kohn, David Williams

Published: November 3, 2025

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Christine Briscoe
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