Molecular remission of infant B-ALL after infusion of universal TALEN gene-edited CAR T cells
Waseem Qasim(Great Ormond Street Hospital), Hong Zhan(University College London), Sujith Samarasinghe(Great Ormond Street Hospital), Stuart Adams(Great Ormond Street Hospital), Persis Amrolia(Great Ormond Street Hospital), Sian Stafford(University College London), Katie Butler(University College London), Christine Rivat(University College London), Gary Wright(Great Ormond Street Hospital), Kathy Somana(Great Ormond Street Hospital), Sara Ghorashian(University College London), Danielle Pinner(Great Ormond Street Hospital), Gul Ahsan(Great Ormond Street Hospital), Kimberly Gilmour(Great Ormond Street Hospital), Giovanna Lucchini(Great Ormond Street Hospital), Sarah Inglott(Great Ormond Street Hospital), William Mifsud(Great Ormond Street Hospital), Robert Chiesa(Great Ormond Street Hospital), Karl S. Peggs(University College London), Lucas Chan(King's College London), Farzin Farzaneh(King's College London), Adrian J. Thrasher(University College London), Ajay Vora(Sheffield Children's Hospital), Martin Pulé(University College London), Paul Veys(University College London)
Cited by 884Open Access
Abstract
B cell acute lymphoblastic leukemia received lymphodepleting chemotherapy and anti-CD52 serotherapy, followed by a single-dose infusion of UCART19 cells. Molecular remissions were achieved within 28 days in both infants, and UCART19 cells persisted until conditioning ahead of successful allogeneic stem cell transplantation. This bridge-to-transplantation strategy demonstrates the therapeutic potential of gene-editing technology.
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