The DOI matches its Crossref record; 34 of the 34 references it cites were found in a registry or an index.
Betibeglogene Autotemcel Gene Therapy for Non–β 0 /β 0 Genotype β-Thalassemia
Open accessAbstract
BACKGROUND: ) gene. METHODS: genotype. Patients underwent myeloablation with busulfan (with doses adjusted on the basis of pharmacokinetic analysis) and received beti-cel intravenously. The primary end point was transfusion independence (i.e., a weighted average hemoglobin level of ≥9 g per deciliter without red-cell transfusions for ≥12 months). RESULTS: ) was 8.7 g per deciliter (range, 5.2 to 10.6) in patients who had transfusion independence. The safety profile of beti-cel was consistent with that of busulfan-based myeloablation. Four patients had at least one adverse event that was considered by the investigators to be related or possibly related to beti-cel; all events were nonserious except for thrombocytopenia (in 1 patient). No cases of cancer were observed. CONCLUSIONS: genotype, including those younger than 12 years of age. (Funded by Bluebird Bio; HGB-207 ClinicalTrials.gov number, NCT02906202.).
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Cites
A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency
Hacein-Bey-Abina, S., von Kalle, C., Schmidt, M., Le Deist, F., Wulffraat, N., McIntyre, E., Radford, I., Villeval, J.-L., Fraser, C. C., Cavazzana-Calvo, M., et al. (2003) · New England Journal of Medicine · doi:10.1056/nejm200301163480314 · Cited by 1,867
Self-citationInsertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1
Hacein-Bey-Abina, S., Garrigue, A., Wang, G. P., Soulier, J., Lim, A., Morillon, E., Clappier, E., Caccavelli, L., Delabesse, E., Beldjord, K., et al. (2008) · Journal of Clinical Investigation · doi:10.1172/jci35700 · Cited by 1,854
Cardiovascular T2-star (T2*) magnetic resonance for the early diagnosis of myocardial iron overload
Anderson, L. (2001) · European Heart Journal · doi:10.1053/euhj.2001.2822 · Cited by 1,682
Dunbar, C. E., High, K. A., Joung, J. K., Kohn, D. B., Ozawa, K., & Sadelain, M. (2018) · Science · doi:10.1126/science.aan4672 · Cited by 1,410
Clinical use of lentiviral vectors
Milone, M. C., & O’Doherty, U. (2018) · Leukemia · doi:10.1038/s41375-018-0106-0 · Cited by 930
Stein, S., Ott, M. G., Schultze-Strasser, S., Jauch, A., Burwinkel, B., Kinner, A., Schmidt, M., Krämer, A., Schwäble, J., Glimm, H., et al. (2010) · Nature Medicine · doi:10.1038/nm.2088 · Cited by 716
Self-citationGene Therapy in Patients with Transfusion-Dependent β-Thalassemia
Thompson, A. A., Walters, M. C., Kwiatkowski, J., Rasko, J. E. J., Ribeil, J.-A., Hongeng, S., Magrin, E., Schiller, G. J., Payen, E., Semeraro, M., et al. (2018) · New England Journal of Medicine · doi:10.1056/nejmoa1705342 · Cited by 684
Self-citationTaher, A. T., Musallam, K. M., & Cappellini, M. D. (2021) · New England Journal of Medicine · doi:10.1056/nejmra2021838 · Cited by 477
Guidelines for the Management of Transfusion Dependent Thalassaemia (TDT)
Cappellini, M. D., Cohen, A. R., Porter, J. B., Taher, A. T., & Viprakasit, V. (2014) · Cited by 400
Self-citationAngelucci, E., Matthes-Martin, S., Baronciani, D., Bernaudin, F., Bonanomi, S., Cappellini, M. D., Dalle, J.-H., Di Bartolomeo, P., de Heredia, C. D., Dickerhoff, R., et al. (2014) · Haematologica · doi:10.3324/haematol.2013.099747 · Cited by 399
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Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia
Locatelli, F., Lang, P., Wall, D., Meisel, R., Corbacioglu, S., Li, A. M., de la Fuente, J., Shah, A. J., Carpenter, B., Kwiatkowski, J. L., et al. (2024) · New England Journal of Medicine · doi:10.1056/nejmoa2309673 · Cited by 253
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Metadata
- Authors
- Locatelli, F., Thompson, A. A., Kwiatkowski, J. L., Porter, J. B., Thrasher, A. J., Hongeng, S., Sauer, M. G., Thuret, I., Lal, A., Algeri, M., Schneiderman, J., Olson, T. S., Carpenter, B., Amrolia, P. J., Anurathapan, U., Schambach, A., Chabannon, C., Schmidt, M., Labik, I., Elliot, H., Guo, R., Asmal, M., Colvin, R. A., & Walters, M. C.
- Venue
- New England Journal of Medicine
- Publisher
- Massachusetts Medical Society
- Published
- 2022-02-03
- Volume
- 386
- Issue
- 5
- Pages
- 415-427
- ISSN
- 0028-4793 (ISSN-L)
- 1533-4406
- OpenAlex
- W4200123108
- Cited by
- 283
- References
- 34
- FWCI
- 17.23
- Type
- journal article
- Language
- en
- Open access
- hybrid
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- Locatelli, F., Thompson, A. A., Kwiatkowski, J. L., Porter, J. B., Thrasher, A. J., Hongeng, S., Sauer, M. G., Thuret, I., Lal, A., Algeri, M., Schneiderman, J., Olson, T. S., Carpenter, B., Amrolia, P. J., Anurathapan, U., Schambach, A., Chabannon, C., Schmidt, M., Labik, I., . . . Walters, M. C. (2022). Betibeglogene Autotemcel Gene Therapy for Non–β 0 /β 0 Genotype β-Thalassemia. New England Journal of Medicine. https://doi.org/10.1056/nejmoa2113206
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