The DOI matches its Crossref record; 18 of the 18 references it cites were found in a registry or an index.
Meganucleases and Other Tools for Targeted Genome Engineering: Perspectives and Challenges for Gene Therapy
Open accessAbstract
The importance of safer approaches for gene therapy has been underscored by a series of severe adverse events (SAEs) observed in patients involved in clinical trials for Severe Combined Immune Deficiency Disease (SCID) and Chromic Granulomatous Disease (CGD). While a new generation of viral vectors is in the process of replacing the classical gamma-retrovirus – based approach, a number of strategies have emerged based on non-viral vectorization and/or targeted insertion aimed at achieving safer gene transfer. Currently, these methods display lower efficacies than viral transduction although many of them can yield more than 1% engineered cells in vitro. Nuclease-based approaches, wherein an endonuclease is used to trigger site-specific genome editing, can significantly increase the percentage of targeted cells. These methods therefore provide a real alternative to classical gene transfer as well as gene editing. However, the first endonuclease to be in clinic today is not used for gene transfer, but to inactivate a gene (CCR5) required for HIV infection. Here, we review these alternative approaches, with a special emphasis on meganucleases, a family of naturally occurring rare-cutting endonucleases, and speculate on their current and future potential. Keywords: Homing endonuclease, Zinc-finger nuclease, Recombinase, Transposons, gene transfer, protein engineering, viral vector, Severe Combined Immune Deficiency Disease, Chromic Granulomatous Disease, transposases, Streptomyces phage, human embryonic stem, pluripotent stem, chimeric antibody receptor, Zinc-finger nucleases, Activator Like Effectors, pseudo-symmetric, meganucleases, protein-protein interactions, single-strand annealing, leukemia, Adeno-Associated Virus, Haematopoietic Stem Cell, Triplex-Forming Oligonucleotides, Zinc-Finger Protein
Overview
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Integrity signals
Registry
Cites
Statistics
Verification
Claims the paper makes
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Self-check
Percentages, sums and reported differences re-computed from the paper’s own numbers.
The self-check needs the paper’s full text.
Citations
Cites
The mechanism of double-strand DNA break repair by the nonhomologous DNA end-joining pathway
Lieber, M. R. (2010) · Annual Review of Biochemistry · doi:10.1146/annurev.biochem.052308.093131 · Cited by 2,882
Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1
Hacein-Bey-Abina, S., Garrigue, A., Wang, G. P., Soulier, J., Lim, A., Morillon, E., Clappier, E., Caccavelli, L., Delabesse, E., Beldjord, K., et al. (2008) · Journal of Clinical Investigation · doi:10.1172/jci35700 · Cited by 1,854
Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy
Cartier, N., Hacein-Bey-Abina, S., Bartholomae, C. C., Veres, G., Schmidt, M., Kutschera, I., Vidaud, M., Abel, U., Dal-Cortivo, L., Caccavelli, L., et al. (2009) · Science · doi:10.1126/science.1171242 · Cited by 1,508
Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia
Cavazzana-Calvo, M., Payen, E., Negre, O., Wang, G., Hehir, K., Fusil, F., Down, J., Denaro, M., Brady, T., Westerman, K., et al. (2010) · Nature · doi:10.1038/nature09328 · Cited by 1,276
An improved zinc-finger nuclease architecture for highly specific genome editing
Miller, J. C., Holmes, M. C., Wang, J., Guschin, D. Y., Lee, Y.-L., Rupniewski, I., Beausejour, C. M., Waite, A. J., Wang, N. S., Kim, K. A., et al. (2007) · Nature Biotechnology · doi:10.1038/nbt1319 · Cited by 1,051
Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleases
Perez, E. E., Wang, J., Miller, J. C., Jouvenot, Y., Kim, K. A., Liu, O., Wang, N., Lee, G., Bartsevich, V. V., Lee, Y.-L., et al. (2008) · Nature Biotechnology · doi:10.1038/nbt1410 · Cited by 999
Lombardo, A., Genovese, P., Beausejour, C. M., Colleoni, S., Lee, Y.-L., Kim, K. A., Ando, D., Urnov, F. D., Galli, C., Gregory, P. D., et al. (2007) · Nature Biotechnology · doi:10.1038/nbt1353 · Cited by 845
Homology-directed repair is a major double-strand break repair pathway in mammalian cells
Liang, F., Han, M., Romanienko, P. J., & Jasin, M. (1998) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.95.9.5172 · Cited by 649
Gene targeting using zinc finger nucleases
Porteus, M. H., & Carroll, D. (2005) · Nature Biotechnology · doi:10.1038/nbt1125 · Cited by 641
Montini, E., Cesana, D., Schmidt, M., Sanvito, F., Bartholomae, C. C., Ranzani, M., Benedicenti, F., Sergi, L. S., Ambrosi, A., Ponzoni, M., et al. (2009) · Journal of Clinical Investigation · doi:10.1172/jci37630 · Cited by 571
Cited by
CRISPR-Cas systems for editing, regulating and targeting genomes
Sander, J. D., & Joung, J. K. (2014) · Nature Biotechnology · doi:10.1038/nbt.2842 · Cited by 3,153
Dunbar, C. E., High, K. A., Joung, J. K., Kohn, D. B., Ozawa, K., & Sadelain, M. (2018) · Science · doi:10.1126/science.aan4672 · Cited by 1,410
CRISPR/Cas9 in Genome Editing and Beyond
Wang, H., La Russa, M., & Qi, L. S. (2016) · Annual Review of Biochemistry · doi:10.1146/annurev-biochem-060815-014607 · Cited by 1,110
Kim, J. Y., Kim, Y.-G., & Lee, G. M. (2012) · Applied Microbiology and Biotechnology · doi:10.1007/s00253-011-3758-5 · Cited by 801
Efficient ablation of genes in human hematopoietic stem and effector cells using CRISPR/Cas9
Mandal, P. K., Ferreira, L. M. R., Collins, R., Meissner, T. B., Boutwell, C. L., Friesen, M., Vrbanac, V., Garrison, B. S., Stortchevoi, A., Bryder, D., et al. (2014) · Cell stem cell · doi:10.1016/j.stem.2014.10.004 · Cited by 475
“spread adoption of these technologies for therapeutic use (Silva et al., 2011).”
Ousterout, D. G., Kabadi, A. M., Thakore, P. I., Majoros, W. H., Reddy, T. E., & Gersbach, C. A. (2015) · Nature Communications · doi:10.1038/ncomms7244 · Cited by 470
Genome engineering empowers the diatom Phaeodactylum tricornutum for biotechnology
Daboussi, F., Leduc, S., Maréchal, A., Dubois, G., Guyot, V., Perez-Michaut, C., Amato, A., Falciatore, A., Juillerat, A., Beurdeley, M., et al. (2014) · Nature Communications · doi:10.1038/ncomms4831 · Cited by 455
Self-citationDNA Repair Pathway Choices in CRISPR-Cas9-Mediated Genome Editing
Xue, C., & Greene, E. C. (2021) · Trends in Genetics · doi:10.1016/j.tig.2021.02.008 · Cited by 431
CRISPR/Cas: A powerful tool for gene function study and crop improvement
Zhang, D., Zhang, Z., Unver, T., & Zhang, B. (2021) · Journal of Advanced Research · doi:10.1016/j.jare.2020.10.003 · Cited by 373
Genome-Editing Technologies: Principles and Applications
Gaj, T., Sirk, S. J., Shui, S.-L., & Liu, J. (2016) · Cold Spring Harbor Perspectives in Biology · doi:10.1101/cshperspect.a023754 · Cited by 381
Ex vivo gene transfer and correction for cell-based therapies
Naldini, L. (2011) · Nature Reviews Genetics · doi:10.1038/nrg2985 · Cited by 358
Holkers, M., Maggio, I., Liu, J., Janssen, J. M., Miselli, F., Mussolino, C., Recchia, A., Cathomen, T., & Gonçalves, M. A. F. V. (2013) · Nucleic Acids Research · doi:10.1093/nar/gks1446 · Cited by 312
Therapeutic glycoprotein production in mammalian cells
Lalonde, M.-E., & Durocher, Y. (2017) · Journal of Biotechnology · doi:10.1016/j.jbiotec.2017.04.028 · Cited by 296
Genome Editing in Human Pluripotent Stem Cells: Approaches, Pitfalls, and Solutions
Hendriks, W. T., Warren, C. R., & Cowan, C. A. (2016) · Cell Stem Cell · doi:10.1016/j.stem.2015.12.002 · Cited by 110
Genome editing and improvement of abiotic stress tolerance in crop plants
Yadav, R. K., Tripathi, M. K., Tiwari, S., Tripathi, N., Asati, R., Chauhan, S., Tiwari, P. N., & Payasi, D. K. (2023) · Life · doi:10.3390/life13071456 · Cited by 75
Towards a new era in medicine: therapeutic genome editing
Porteus, M. H. (2015) · Genome Biology · doi:10.1186/s13059-015-0859-y · Cited by 69
Customizing the genome as therapy for the β-hemoglobinopathies.
Canver, M. C., & Orkin, S. H. (2016) · Blood · doi:10.1182/blood-2016-01-678128 · Cited by 61
Latest Trends and Developments in Microalgae as Potential Source for Biofuels: The Case of Diatoms
Mourya, M., Khan, M. J., Ahirwar, A., Schoefs, B., Marchand, J., Rai, A., Varjani, S., Rajendran, K., Banu, J. R., & Vinayak, V. (2022) · Fuel · doi:10.1016/j.fuel.2021.122738 · Cited by 52
Gene Therapy for Inherited Retinal Degeneration
Arbabi, A., Liu, A., & Ameri, H. (2019) · Journal of Ocular Pharmacology and Therapeutics · doi:10.1089/jop.2018.0087 · Cited by 30
Genome Editing for the β-Hemoglobinopathies
Porteus, M. H. (2017) · Advances in Experimental Medicine and Biology · doi:10.1007/978-1-4939-7299-9_8 · Cited by 8
Evolution of prime editing systems: move forward to the treatment of hereditary diseases
Volodina, O. V., Fabrichnikova, A. R., Anuchina, A. A., Mishina, O. S., Lavrov, A. V., & Smirnikhina, S. A. (2025) · Current Gene Therapy · doi:10.2174/0115665232295117240405070809 · Cited by 1
Metadata
- Authors
- Silva, G., Poirot, L., Galetto, R., Smith, J., Montoya, G., Duchateau, P., & Paques, F.
- Venue
- Current Gene Therapy
- Publisher
- Bentham Science Publishers Ltd.
- Published
- 2011-02-01
- Volume
- 11
- Issue
- 1
- Pages
- 11-27
- ISSN
- 1566-5232 (ISSN-L)
- OpenAlex
- W2142068612
- Cited by
- 410
- References
- 18
- FWCI
- 9.29
- Type
- journal article
- Language
- en
- Open access
- green
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
Are you an author, or do you see a problem with this page? Request a review
Data sources
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.
