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]
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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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