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
Erica B. Esrick(Broad Institute), David A. Williams(Harvard University), Jerome Ritz(Harvard University), Kit L. Shaw(Boston Children's Hospital), Aoife Doto(University of Pennsylvania), J.K. Everett(University of Pennsylvania), Myriam Armant(Dana-Farber/Boston Children's Cancer and Blood Disorders Center), Frederic D. Bushman(University of Pennsylvania), Akshat Jain(Loma Linda University Children's Hospital), David G. Justus(Boston Children's Hospital), Satiro De Oliveira(University of California, Los Angeles), Heather Daley(Dana-Farber Cancer Institute), Gavin D. Roach(University of California, Los Angeles), John Manis(Broad Institute), Donald B. Kohn(University of California, Los Angeles), Pei‐Chi Kao(Boston Children's Hospital), Theodore B. Moore(University of California, Los Angeles), O. B. da Silva(Boston Children's Hospital), Colleen Dansereau(Dana-Farber/Boston Children's Cancer and Blood Disorders Center), Boya Liu(Boston Children's Hospital), Hélène Negre(Dana-Farber Cancer Institute), Amy Federico(Boston Children's Hospital), Wendy B. London(Dana-Farber Cancer Institute), Helene Vincon(Boston Children's Hospital), Leslie Lehmann(Harvard University)
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