The DOI matches its Crossref record; 7 of the 7 references it cites were found in a registry or an index.
Acute Myeloid Leukemia Case after Gene Therapy for Sickle Cell Disease
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“Although insertional mutagenesis risk has been reduced by improved vector design19, the development of myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) at 3–5.5 years post-transplantation in 2 of 47 patients who had undergone GT for SCD20–23 has generated renewed concerns.”
From Clonal selection of hematopoietic stem cells after gene therapy for sickle cell disease (2023) · cites this paper as [21]
In the citing paper
“though neither were reported to be mutated in patients who experienced post-GT myeloid malignancies20,21”
From Clonal selection of hematopoietic stem cells after gene therapy for sickle cell disease (2023) · cites this paper as [21]
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Kato, G. J., Piel, F. B., Reid, C. D., Gaston, M. H., Ohene-Frempong, K., Krishnamurti, L., Smith, W. R., Panepinto, J. A., Weatherall, D. J., Costa, F. F., et al. (2018) · Nature Reviews Disease Primers · doi:10.1038/nrdp.2018.10 · Cited by 1,501
Pathophysiology of Sickle Cell Disease
Sundd, P., Gladwin, M. T., & Novelli, E. M. (2019) · Annual Review of Pathology Mechanisms of Disease · doi:10.1146/annurev-pathmechdis-012418-012838 · Cited by 813
Gene therapy in a patient with sickle cell disease
Ribeil, J.-A., Hacein-Bey-Abina, S., Payen, E., Magnani, A., Semeraro, M., Magrin, E., Caccavelli, L., Neven, B., Bourget, P., El Nemer, W., et al. (2017) · New England Journal of Medicine · doi:10.1056/nejmoa1609677 · Cited by 696
Gene 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
Biologic and Clinical Efficacy of LentiGlobin for Sickle Cell Disease
Kanter, J., Walters, M. C., Krishnamurti, L., Mapara, M. Y., Kwiatkowski, J. L., Rifkin-Zenenberg, S., Aygun, B., Kasow, K. A., Pierciey, F. J., Bonner, M., et al. (2022) · New England Journal of Medicine · doi:10.1056/nejmoa2117175 · Cited by 442
Self-citationHigh-resolution insertion-site analysis by linear amplification-mediated PCR (LAM-PCR)
Schmidt, M., Schwarzwaelder, K., Bartholomae, C., Zaoui, K., Ball, C., Pilz, I., Braun, S., Glimm, H., & von Kalle, C. (2007) · Nature Methods · doi:10.1038/nmeth1103 · Cited by 328
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Cited by
Exagamglogene Autotemcel for Severe Sickle Cell Disease
Frangoul, H., Locatelli, F., Sharma, A., Bhatia, M., Mapara, M., Molinari, L., Wall, D., Liem, R. I., Telfer, P., Shah, A. J., et al. (2024) · New England Journal of Medicine · doi:10.1056/nejmoa2309676 · Cited by 493
Biologic and Clinical Efficacy of LentiGlobin for Sickle Cell Disease
Kanter, J., Walters, M. C., Krishnamurti, L., Mapara, M. Y., Kwiatkowski, J. L., Rifkin-Zenenberg, S., Aygun, B., Kasow, K. A., Pierciey, F. J., Bonner, M., et al. (2022) · New England Journal of Medicine · doi:10.1056/nejmoa2117175 · Cited by 442
Self-citationTreatment of cerebral adrenoleukodystrophy: allogeneic transplantation and lentiviral gene therapy
Gupta, A. O., Raymond, G., Pierpont, E. I., Kemp, S., McIvor, R. S., Rayannavar, A., Miller, B., Lund, T. C., & Orchard, P. J. (2022) · Expert Opinion on Biological Therapy · doi:10.1080/14712598.2022.2124857 · Cited by 67
“after treatment on a trial utilizing the BB305 lentiviral vector encoding the -globin gene (HGB-206 Study) [85,86].”
BackgroundMethodClonal selection of hematopoietic stem cells after gene therapy for sickle cell disease
Spencer Chapman, M., Cull, A. H., Ciuculescu, M. F., Esrick, E. B., Mitchell, E., Jung, H., O’Neill, L., Roberts, K., Fabre, M. A., Williams, N., et al. (2023) · Nature Medicine · doi:10.1038/s41591-023-02636-6 · Cited by 67
Ex vivo modification of hematopoietic stem and progenitor cells for gene therapy
Williams, D. A., Kohn, D. B., & Thrasher, A. J. (2025) · Molecular Therapy · doi:10.1016/j.ymthe.2025.03.058 · Cited by 11
Metadata
- Authors
- Goyal, S., Tisdale, J., Schmidt, M., Kanter, J., Jaroscak, J., Whitney, D., Bitter, H., Gregory, P. D., Parsons, G., Foos, M., Yeri, A., Gioia, M., Voytek, S. B., Miller, A., Lynch, J., Colvin, R. A., & Bonner, M.
- Venue
- New England Journal of Medicine
- Publisher
- Massachusetts Medical Society
- Published
- 2022-01-13
- Volume
- 386
- Issue
- 2
- Pages
- 138-147
- ISSN
- 0028-4793
- 1533-4406
- Cited by
- 211
- References
- 7
- Language
- en
- Funders
- bluebird bio
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- Goyal, S., Tisdale, J., Schmidt, M., Kanter, J., Jaroscak, J., Whitney, D., Bitter, H., Gregory, P. D., Parsons, G., Foos, M., Yeri, A., Gioia, M., Voytek, S. B., Miller, A., Lynch, J., Colvin, R. A., & Bonner, M. (2022). Acute Myeloid Leukemia Case after Gene Therapy for Sickle Cell Disease. New England Journal of Medicine. https://doi.org/10.1056/nejmoa2109167
