Journal
SCIENCE TRANSLATIONAL MEDICINE
Volume 9, Issue 374, Pages -Publisher
AMER ASSOC ADVANCEMENT SCIENCE
DOI: 10.1126/scitranslmed.aaj2013
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Funding
- NIH Research (NIHR)
- European Union FP7 [602239]
- NIHR [RP-2014-05-007]
- Wellcome Trust [104807/Z/14/Z]
- Cellectis SA [WO 02013176915]
- BBSRC [BB/N003853/1, BB/E005896/1] Funding Source: UKRI
- Biotechnology and Biological Sciences Research Council [BB/N003853/1, BB/E005896/1] Funding Source: researchfish
- Great Ormond Street Hospital Childrens Charity [W1105] Funding Source: researchfish
- National Institute for Health Research [NIHR-RP-R3-12-001, RP-2014-05-007] Funding Source: researchfish
- Wellcome Trust [104807/Z/14/Z] Funding Source: researchfish
- National Institutes of Health Research (NIHR) [NIHR-RP-R3-12-001] Funding Source: National Institutes of Health Research (NIHR)
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Autologous T cells engineered to express chimeric antigen receptor against the B cell antigen CD19 (CAR19) are achieving marked leukemic remissions in early-phase trials but can be difficult to manufacture, especially in infants or heavily treated patients. We generated universal CAR19 (UCART19) T cells by lentiviral transduction of non-human leukocyte antigen-matched donor cells and simultaneous transcription activator-like effector nuclease (TALEN)-mediated gene editing of T cell receptor a chain and CD52 gene loci. Two infants with relapsed refractory CD19(+) 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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