Highly efficient collection and manufacture of autologous HSC gene therapy cell product for patients with sickle cell disease using a lentiviral vector containing a shmir targeting BCL11a

John Manis(Broad Institute), David A. Williams(Boston Children's Hospital), Erica B. Esrick(Broad Institute), Leslie Lehmann(Harvard University), Jerome Ritz(Dana-Farber Cancer Institute), Neena Kapoor(St Vincent's Hospital Sydney), Kit L. Shaw(Boston Children's Hospital), Edmund K. Waller(Emory University), Myriam Armant(Dana-Farber/Boston Children's Cancer and Blood Disorders Center), Ellen Proeung(Boston Children's Hospital), Mehrdad Abedi(UC Davis Comprehensive Cancer Center), Carly Howard(Boston Children's Hospital), Akshat Jain(Loma Linda University), David G. Justus(Boston Children's Hospital), Gary J. Schiller(Fred Hutch Cancer Center), Angela Treml(Medical College of Wisconsin), Mary Eapen(Medical College of Wisconsin), Heather Daley(Dana-Farber Cancer Institute), Donald B. Kohn(University of California, Los Angeles), Joseph H. Antin(Dana-Farber Cancer Institute), Leah Cheng(Boston Children's Hospital), Colleen Dansereau(Dana-Farber/Boston Children's Cancer and Blood Disorders Center), Jeanne E. Hendrickson(Emory University), Aimee Sarnie(Boston Children's Hospital), Amy Federico(Boston Children's Hospital), Mark Walters(University of California, San Francisco), Sonali Chaudhury(Lurie Children's Hospital), Mary Horowitz(Medical College of Wisconsin), Victoria H. Coleman‐Cowger(University of Maryland, Baltimore), Helene Vincon(Boston Children's Hospital), Michael A. Pulsipher(Primary Children's Hospital)
Blood
November 3, 2025
Cited by 1


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