The DOI matches its Crossref record; 149 of the 149 references it cites were found in a registry or an index.
Meganucleases and DNA Double-Strand Break-Induced Recombination: Perspectives for Gene Therapy
Abstract
Meganucleases are sequence-specific endonucleases recognizing large (>12 bp) sequence sites and several laboratories have used these proteins to induce highly efficient gene targeting in mammalian cells. The recent development of artificial endonucleases with tailored specificities has opened the door for a wide range of new applications, including therapeutic ones: redesigned endonucleases cleaving chosen sequences could be used to in gene therapy to correct mutated genes or introduce transgenes in chosen loci. Such "targeted" approaches markedly differ from current gene therapy strategies based on the random insertion of a complementing virus-borne transgene. As a consequence, they should bypass the odds of random insertion. Artificial fusion proteins including Zinc-Finger binding domains have provided important proofs of concept, however the toxicity of these proteins is still an issue. Today custom-designed homing endonucleases, the natural meganucleases, could represent an efficient alternative. After a brief description of the origin of the technology, current systems based on redesigned endonucleases will be presented, with a special emphasis on the recent advances in homing endonuclease engineering. Finally, we will discuss the main issues that will need to be addressed in order to bring this promising technology to the patient.
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Citations
Cites
LMO2 -Associated Clonal T Cell Proliferation in Two Patients after Gene Therapy for SCID-X1
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The double-strand-break repair model for recombination
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Isolation and characterization
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Multiple Pathways of Recombination Induced by Double-Strand Breaks in Saccharomyces cerevisiae
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Self-citationZinc Finger-DNA Recognition: Crystal Structure of a Zif268-DNA Complex at 2.1 Å
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Cited by
Genome Engineering With Zinc-Finger Nucleases
Carroll, D. (2011) · Genetics · doi:10.1534/genetics.111.131433 · Cited by 1,076
Precision genome engineering and agriculture: opportunities and regulatory challenges
Voytas, D. F., & Gao, C. (2014) · PLoS Biology · doi:10.1371/journal.pbio.1001877 · Cited by 458
Safe harbours for the integration of new DNA in the human genome
Sadelain, M., Papapetrou, E. P., & Bushman, F. D. (2012) · Nature Reviews Cancer · doi:10.1038/nrc3179 · Cited by 441
Viral vectors: from virology to transgene expression
Bouard, D., Alazard‐Dany, N., & Cosset, F. (2009) · British Journal of Pharmacology · doi:10.1038/bjp.2008.349 · Cited by 360
Tracking genome engineering outcome at individual DNA breakpoints
Certo, M. T., Ryu, B. Y., Annis, J. E., Garibov, M., Jarjour, J., Rawlings, D. J., & Scharenberg, A. M. (2011) · Nature Methods · doi:10.1038/nmeth.1648 · Cited by 318
Metadata
- Authors
- Paques, F., & Duchateau, P.
- Venue
- Current Gene Therapy
- Publisher
- Bentham Science Publishers Ltd.
- Published
- 2007-02-01
- Volume
- 7
- Issue
- 1
- Pages
- 49-66
- ISSN
- 1566-5232 (ISSN-L)
- OpenAlex
- W2024533323
- Cited by
- 273
- References
- 149
- FWCI
- 7.30
- Type
- journal article
- Language
- en
- Open access
- closed
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- Paques, F., & Duchateau, P. (2007). Meganucleases and DNA Double-Strand Break-Induced Recombination: Perspectives for Gene Therapy. Current Gene Therapy. https://doi.org/10.2174/156652307779940216
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Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).
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