CORRECTION article

Front. Genome Ed., 17 May 2021

Sec. Genome Editing in Blood Disorders

Volume 3 - 2021 | https://doi.org/10.3389/fgeed.2021.682171

Corrigendum: Targeted Gene Delivery: Where to Land

  • INTEGRARE, UMR_S951, Genethon, Inserm, Univ Evry, Univ Paris-Saclay, Evry, France

In the original article, there was a mistake in Table 1 as published. The references indicated in row B are wrong. The corrected Table 1 appears in the attached below.

Table 1

Integration strategiesAdvantagesDisadvantagesReferences
AEndogenous locusPhysiological transgene expression
Corrects multiple mutations
Gene-specific strategy
Limited to gene body mutations
Urnov et al., ; Lombardo et al., ; Li et al., ; Genovese et al., ; Voit et al., ; Dever et al., ; Hubbard et al., ; Schiroli et al., ; Sweeney et al., ; Kuo et al., ; Wang et al., ; Rai et al., ; Wang L. et al.,
BSuperactive promoters (ALB, HBA)Accommodates different transgenes
Supraphysiological expression
Few integrations required
Partial gene disruption
Limited to non-cell autonomous disorders
Extensive validation required
Barzel et al., ; Sharma et al., ; Davidoff and Nathwani, ; Laoharawee et al., ; Chen et al., ; Conway et al., ; De Caneva et al., ; Ou et al., , ; Zhang et al., ; Wang Q. et al.,
CTolerant to integration (AAVS1, CCR5, Rosa26)Accommodates different transgenesArtificial promoters required
Variable expression
De Ravin et al., ; Diez et al., ; Stephens et al., , ; Gomez-Ospina et al., ; Scharenberg et al.,
DChromatin domains (NAD)Fine gene regulation
Far from oncogenic genes
No proof-of-principle in clinically relevant modelsSchenkwein et al.,
EDisease-modifier genes (CCR5, HBA)Improve therapeutic effect
Lower therapeutic threshold
Extensive validation required
Limited to well-known diseases
Voit et al., ; Wiebking et al.,
FSpecificity
Exchange
(TCR, BCR)
Improved CAR expression and potencyOff-targets
Translocations risk (for multiple edits)
Eyquem et al., ; MacLeod et al., ; Greiner et al., ; Hartweger et al., ; Moffett et al., ; Voss et al.,

(A–F) The advantages and disadvantages of different integration strategies.

Scissors: nuclease; Solid arrows: promoters; Enh, enhancers; TAD, topologically associating; d, domain; Solid ovals: histone modifications; Solid squares: DNA modifications.

The authors apologize for this error and state that this does not change the scientific conclusions of the article in any way. The original article has been updated.

References

Summary

Keywords

genome editing, gene therapy, nuclease, CRISPR, targeted integration (TI), knock-in, safe harbor, homologous recombination (HR)

Citation

Pavani G and Amendola M (2021) Corrigendum: Targeted Gene Delivery: Where to Land. Front. Genome Ed. 3:682171. doi: 10.3389/fgeed.2021.682171

Received

17 March 2021

Accepted

06 April 2021

Published

17 May 2021

Volume

3 - 2021

Edited and reviewed by

Pietro Genovese, Boston Children's Hospital and Harvard Medical School, United States

Updates

Copyright

*Correspondence: Mario Amendola

†Present address: Giulia Pavani, The Children's Hospital of Philadelphia, Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Philadelphia, PA, United States

This article was submitted to Genome Editing in Blood Disorders, a section of the journal Frontiers in Genome Editing

Disclaimer

All claims expressed in this article are solely those of the authors and do not necessarily represent those of their affiliated organizations, or those of the publisher, the editors and the reviewers. Any product that may be evaluated in this article or claim that may be made by its manufacturer is not guaranteed or endorsed by the publisher.

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