Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery

By A. Lombardo, P. Genovese, C. Beauséjour, S. Colleoni, Ya-Li Lee, Kenneth A Kim, D. Ando, F. Urnov, C. Galli, P. Gregory and 2 more. Nature Biotechnology, 2007

916 citations recorded by the source corpus.

Subfields: Genetics, Molecular Biology

View Paper: https://www.semanticscholar.org/paper/55ce5845bb8720d0a808a15dd75b7c63f37ea76d

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In Genetics

1. Feng Zhang
2. P. Hsu
3. Eric S. Lander
4. B. Davidson
5. Jennifer A. Doudna
6. Lei S. Qi
7. Haifeng Wang
8. Marie La Russa

In Molecular Biology

1. Feng Zhang
2. P. Hsu
3. Eric S. Lander
4. P. McCray
5. B. Davidson
6. J. Keith Joung
7. Jennifer A. Doudna
8. Lei S. Qi

Citations in the corpus

• A novel TALE nuclease scaffold enables high genome editing activity in combination with low toxicity (2011, background): “The target sites and the recognition a-helices of the EGFP (17), CCR5 (9) and IL2RG-specific (36) ZFNs have been described.”
• Rapid “open-source” engineering of customized zinc-finger nucleases for highly efficient gene modification (2008, neutral): “To test whether OPEN ZFNs induce mutations by NHEJ at endogenous human genes, we employed a CEL I nuclease-based mutation detection assay (Figure 3A) (Lombardo et al., 2007; Miller et al., 2007 We also tested whether OPEN ZFNs induced mutations in endogenous plant genes.”
• Current prospects for RNA interference-based therapies (2011, background): “Like adenoviruses and AAVs, the vector tropism of lentiviruses can be altered, in this case through manipulation of the viral envelope used in vector production 88 .”
• Development and Applications of CRISPR-Cas9 for Genome Engineering (2014, background): “For example, NHEJ-mediated inactivation of the CCR5 receptor in lymphocytes (Lombardo et al., 2007) may be a viable strategy for circumventing HIV infection, whereas deletion of PCSK9 (Cohen et al., 2005) or angiopoietin (Musunuru et al., 2010) may provide therapeutic effects against…”
• Zinc finger nuclease-mediated CCR5 knockout hematopoietic stem cell transplantation controls HIV-1 in vivo (2010, neutral): “Since DSB formation requires only transient expression of ZFNs, gene delivery methods that are suitable to express ZFNs include plasmid DNA nucleofection, integrase-defective lentiviral vectors and adenoviral vectors (16,19,21).”