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Lentiviral gene therapy with reduced-intensity conditioning for sickle cell disease: a phase 1/2 trial

Michael Grimley, Stella M. Davies, Archana Shrestha, Amy Shova, Monika Asnani, Michael Kent, Farzana Sayani, Charles T. Quinn, Omar Niss, Carolyn Lutzko, Parinda A. Mehta, Pooja Khandelwal, Courtney Little, Sharat Chandra, Sydney Felker, Mengna Chi, Theodosia A. Kalfa, Jennifer Knight‐Madden, Paritha Arumugam, Kristie N. Ramos, Scott R. Witting, Teresa Latham, Frederic D. Bushman, Punam Malik

2025Nature Medicine15 citationsDOIOpen Access PDF

Topics & Concepts

Genetic enhancementMedicineConditioningDiseaseClinical trialOncologyImmunologyInternal medicineGeneBiologyGeneticsStatisticsMathematicsHemoglobinopathies and Related DisordersVirus-based gene therapy researchParvovirus B19 Infection Studies
Lentiviral gene therapy with reduced-intensity conditioning for sickle cell disease: a phase 1/2 trial | Litcius