The DOI matches its Crossref record; 2 of the 2 references it cites were found in a registry or an index.
Balancing efficacy and safety in lentiviral vector-mediated hematopoietic stem cell gene therapy
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“No adverse events associated with vector insertion have been reported to date and, although the lentiviral configuration is very similar to that used in cALD, the striking difference is that an internal cellular promoter sequence (derived from the human PGK gene) was used to direct ubiquitous gene expression.”
From Ex vivo modification of hematopoietic stem and progenitor cells for gene therapy (2025) · cites this paper as [60]
In the citing paper
“In a long-term follow-up study of 29 pediatric patients treated with atidarsagene autotemcel, an approved product in the United States and Europe (Lenmeldy), 26 patients were alive with a median follow-up of 3.16 years (range, 0.64-7.51years), and the majority recovered or experienced stabilization in motor function and displayed normal cognitive development.60”
From Ex vivo modification of hematopoietic stem and progenitor cells for gene therapy (2025) · cites this paper as [60]
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Citations
Cites
Uncovering and Dissecting the Genotoxicity of Self-inactivating Lentiviral Vectors In Vivo
Cesana, D., Ranzani, M., Volpin, M., Bartholomae, C., Duros, C., Artus, A., Merella, S., Benedicenti, F., Sergi Sergi, L., Sanvito, F., et al. (2014) · Molecular Therapy · doi:10.1038/mt.2014.3 · Cited by 189
Self-citationHematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy
Duncan, C. N., Bledsoe, J. R., Grzywacz, B., Beckman, A., Bonner, M., Eichler, F. S., Kühl, J.-S., Harris, M. H., Slauson, S., Colvin, R. A., et al. (2024) · New England Journal of Medicine · doi:10.1056/nejmoa2405541 · Cited by 164
Research works printed but not linked: 12
- Ferrari, 2021, Gene therapy using haematopoietic stem and progenitor cells, Nat. Rev. Genet., 22, 216, 10.1038/s41576-020-00298-5 · DOI
- Ferrari, 2023, Genetic engineering meets hematopoietic stem cell biology for next-generation gene therapy, Cell Stem Cell, 30, 549, 10.1016/j.stem.2023.04.014 · DOI
- Calabria, 2024, Long-term lineage commitment in hematopoietic stem cell gene therapy, Nature, 636, 162, 10.1038/s41586-024-08250-x · DOI
- Masiuk, 2024, Ex Vivo Lentiviral Hematopoietic Stem Cell (HSC) Gene Therapy May Represent a Curative Therapy for the Life-Threatening Inborn Error of Immunity Severe Combined Immunodeficiency Due to Adenosine Deaminase (ADA) Deficiency (ADA-SCID), Blood, 144, 10.1182/blood-2024-202543 · DOI
- Cesana, 2012, Whole transcriptome characterization of aberrant splicing events induced by lentiviral vector integrations, J. Clin. Invest., 122, 1667, 10.1172/JCI62189 · DOI
- De Ravin, 2022, Lentivector cryptic splicing mediates increase in CD34+ clones expressing truncated HMGA2 in human X-linked severe combined immunodeficiency, Nat. Commun., 13, 3710, 10.1038/s41467-022-31344-x · DOI · Paper page
- Goyal, 2022, Acute Myeloid Leukemia Case after Gene Therapy for Sickle Cell Disease, N. Engl. J. Med., 386, 138, 10.1056/NEJMoa2109167 · DOI · Paper page
- Jones, 2021, Leukemia after gene therapy for sickle cell disease: insertional mutagenesis, busulfan, both, or neither, Blood, 138, 942, 10.1182/blood.2021011488 · DOI
- Challita, 1995, Multiple modifications in cis elements of the long terminal repeat of retroviral vectors lead to increased expression and decreased DNA methylation in embryonic carcinoma cells, J. Virol., 69, 748, 10.1128/jvi.69.2.748-755.1995 · DOI
- Dunbar, 2024, Weighing the Risks of Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy, N. Engl. J. Med., 391, 1358, 10.1056/NEJMe2409399 · DOI
- Reinhardt, 2021, Long-term outcomes after gene therapy for adenosine deaminase severe combined immune deficiency, Blood, 138, 1304, 10.1182/blood.2020010260 · DOI
- Biffi, 2013, Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy, Science (New York, NY), 341, 1233158, 10.1126/science.1233158 · DOI · Paper page
Cited by
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
Self-citation
Metadata
- Authors
- Montini, E., Naldini, L., Booth, C., Kohn, D. B., & Aiuti, A.
- ORCID
- Montini, Eugenio
- Venue
- Molecular Therapy
- Publisher
- Elsevier BV
- Published
- 2025-01
- Volume
- 33
- Issue
- 1
- Pages
- 6-8
- ISSN
- 1525-0016
- Cited by
- 18
- References
- 2
- Language
- en
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- CoChat. (2026). Citation check: Balancing efficacy and safety in lentiviral vector-mediated hematopoietic stem cell gene therapy. https://app.cochat.ai/papers/10.1016/j.ymthe.2024.12.028
- The paper
- Montini, E., Naldini, L., Booth, C., Kohn, D. B., & Aiuti, A. (2025). Balancing efficacy and safety in lentiviral vector-mediated hematopoietic stem cell gene therapy. Molecular Therapy. https://doi.org/10.1016/j.ymthe.2024.12.028
