Molecular remission of infant B-ALL after infusion of universal TALEN gene-edited CAR T cells
Open accessAbstract
Universal gene-edited CAR19 T cells eliminate infant leukemia.
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Engineered DNA-cleaving enzymes including ZFNs and TALENs demonstrated the promise of therapeutic genome editing.
“Engineered DNA-cleaving enzymes including zinc-finger nucleases (ZFNs) and transcription activator-like effector nucleases (TALENs) demonstrated the promise of therapeutic genome editing.”
From The promise and challenge of therapeutic genome editing (2020) · cites this paper as [19]
The Introduction reports first-in-human use of TALEN-edited T cells, and the Discussion says this application demonstrates the potential of gene-editing strategies for engineered cell therapies. The paper supports the claim about TALENs, but the passages do not establish the same point for ZFNs. (LLM, conf=0.88)
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- Authors
- Qasim, W., Zhan, H., Samarasinghe, S., Adams, S., Amrolia, P., Stafford, S., Butler, K., Rivat, C., Wright, G., Somana, K., Ghorashian, S., Pinner, D., Ahsan, G., Gilmour, K., Lucchini, G., Inglott, S., Mifsud, W., Chiesa, R., Peggs, K. S., Chan, L., Farzaneh, F., Thrasher, A. J., Vora, A., Pule, M., & Veys, P.
- Affiliations
- National Health Service (GB)
- University College London (GB)
- King's College London (GB)
- Sheffield Children's Hospital (GB)
- Cancer Institute, University College London, London WC1E 6DD, UK.
- Corresponding author
- Qasim, Waseem
- ORCID
- Venue
- Science Translational Medicine
- Published
- 2017-01-25
- Volume
- 9
- Issue
- 374
- ISSN
- 1946-6234 (ISSN-L)
- 1946-6242
- OpenAlex
- W2583070206
- Cited by
- 902
- References
- 26
- Citations by year
- 2026: 45 · 2025: 60 · 2024: 69 · 2023: 75 · 2022: 102 · 2021: 111 · 2020: 142 · 2019: 114 · 2018: 113 · 2017: 68 · 2013: 1 · 2012: 1
- FWCI
- 52.67
- Type
- journal article
- Language
- en
- Open access
- green
- discovery.ucl.ac.uk
- Locations
- Science Translational Medicine (publishedVersion)
- PubMed (publishedVersion)
- UCL Discovery (University College London) (submittedVersion)
- Funders
- European Union FP7 (602239)
- NIHR (RP-2014-05-007)
- Wellcome Trust (104807/Z/14/Z)
- Topics
- Health Sciences › Medicine › Oncology › CAR-T cell therapy research
- Health Sciences › Medicine › Public Health, Environmental and Occupational Health › Acute Lymphoblastic Leukemia research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › CRISPR and Genetic Engineering
- Keywords
- B-cell acute lymphoblastic leukemia, infant leukemia, chimeric antigen receptor T cells, TALEN gene editing, CD19, molecular remission, bridge to transplant, allogeneic stem cell transplantation, lymphodepleting chemotherapy
- MeSH terms
- Transcription Activator-Like Effector Nucleases, Transcription Activator-Like Effectors, Gene Editing, CD52 Antigen, Alemtuzumab, Female, Humans, Infant, Neoplasm Recurrence, Local, Receptors, Antigen, T-Cell, Remission Induction, T-Lymphocytes, Transplantation, Homologous, Precursor B-Cell Lymphoblastic Leukemia-Lymphoma, Lentivirus, Hematopoietic Stem Cell Transplantation, Antigens, CD19, Stem Cell Transplantation, Compassionate Use Trials
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