The DOI matches its Crossref record; 164 of the 164 references it cites were found in a registry or an index.
Ex vivo gene transfer and correction for cell-based therapies
Abstract
Cell-based therapies are fast-growing forms of personalized medicine that make use of the steady advances in stem cell manipulation and gene transfer technologies. In this Review, I highlight the latest developments and the crucial challenges for this field, with an emphasis on haematopoietic stem cell gene therapy, which is taken as a representative example given its advanced clinical translation. New technologies for gene correction and targeted integration promise to overcome some of the main hurdles that have long prevented progress in this field. As these approaches marry with our growing capacity for genetic reprogramming of mammalian cells, they may fulfil the promise of safe and effective therapies for currently untreatable diseases.
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“Gene therapy with autologous hematopoietic stem cells has been investigated as an alternative to allogeneic hematopoietic stem-cell transplantation.11–13”
From Hematopoietic stem-cell gene therapy for cerebral adrenoleukodystrophy (2017) · cites this paper as [11]
Citations
Cites
Fate Mapping Analysis Reveals That Adult Microglia Derive from Primitive Macrophages
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In Vivo Gene Delivery and Stable Transduction of Nondividing Cells by a Lentiviral Vector
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Self-citationThe DNA Damage Response: Making It Safe to Play with Knives
Ciccia, A., & Elledge, S. J. (2010) · Molecular Cell · doi:10.1016/j.molcel.2010.09.019 · Cited by 4,368
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Hematopoietic Stem-Cell Transplantation
Copelan, E. A. (2006) · New England Journal of Medicine · doi:10.1056/nejmra052638 · Cited by 2,522
Genome editing with engineered zinc finger nucleases
Urnov, F. D., Rebar, E. J., Holmes, M. C., Zhang, H. S., & Gregory, P. D. (2010) · Nature Reviews Genetics · doi:10.1038/nrg2842 · Cited by 2,285
Natural Innate and Adaptive Immunity to Cancer
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A TALE nuclease architecture for efficient genome editing
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Targeting DNA Double-Strand Breaks with TAL Effector Nucleases
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Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1
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Cited by
Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy
Biffi, A., Montini, E., Lorioli, L., Cesani, M., Fumagalli, F., Plati, T., Baldoli, C., Martino, S., Calabria, A., Canale, S., et al. (2013) · Science · doi:10.1126/science.1233158 · Cited by 1,180
Self-citationGene therapy returns to centre stage
Naldini, L. (2015) · Nature · doi:10.1038/nature15818 · Cited by 1,184
Self-citationLentiviral Hematopoietic Stem Cell Gene Therapy in Patients with Wiskott-Aldrich Syndrome
Aiuti, A., Biasco, L., Scaramuzza, S., Ferrua, F., Cicalese, M. P., Baricordi, C., Dionisio, F., Calabria, A., Giannelli, S., Castiello, M. C., et al. (2013) · Science · doi:10.1126/science.1233151 · Cited by 1,025
Self-citationTherapeutic in vivo gene transfer for genetic disease using AAV: Progress and challenges
Mingozzi, F., & High, K. A. (2011) · Nature Reviews Genetics · doi:10.1038/nrg2988 · Cited by 893
In vitro and ex vivo strategies for intracellular delivery
Stewart, M. P., Sharei, A., Ding, X., Sahay, G., Langer, R., & Jensen, K. F. (2016) · Nature · doi:10.1038/nature19764 · Cited by 885
Metadata
- Authors
- Naldini, L.
- Venue
- Nature Reviews Genetics
- Published
- 2011-03-29
- Volume
- 12
- Issue
- 5
- Pages
- 301-315
- ISSN
- 1471-0056
- 1471-0064
- Cited by
- 358
- References
- 164
- Language
- en
- Licence
- www.springer.com/tdm (tdm)
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Bibliographic data: Semantic Scholar, OpenAlex, Crossref.
Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).
Semantic Scholar’s data is described in: Kinney, R., et al. (2023). The Semantic Scholar Open Data Platform. arXiv:2301.10140.
