Skip to main content

Advertisement

Log in

Selection by essential-gene exon knock-in for the generation of efficient cell therapies

  • Research Briefing
  • Published:

From Nature Biotechnology

View current issue Submit your manuscript

By linking transgene expression with that of a housekeeping gene, SLEEK (selection by essential-gene exon knock-in) enables efficient knock-in of complex cargos in a variety of clinically relevant cell types. Using SLEEK, we were able to substantially improve the tumor suppression ability and in vivo persistence of a cell therapy.

This is a preview of subscription content, log in via an institution to check access.

Access this article

Price excludes VAT (USA)
Tax calculation will be finalised during checkout.

Instant access to the full article PDF.

Fig. 1: SLEEK achieves high transgene knock-in efficiency.

References

  1. Wilkinson, A. C., Igarashi, K. J. & Nakauchi, H. Haematopoietic stem cell self-renewal in vivo and ex vivo. Nat. Rev. Genet. 21, 541–554 (2020). This review article highlights the opportunities for cell therapies applied to hemoglobinopathies.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  2. Bailey, S. R. & Maus, M. V. Gene editing for immune cell therapies. Nat. Biotechnol. 37, 1425–1434 (2019). This review article presents the synchrony between gene editing and cell therapies.

    Article  CAS  PubMed  Google Scholar 

  3. Zhang, L. et al. AsCas12a ultra nuclease facilitates the rapid generation of therapeutic cell medicines. Nat. Commun. 12, 3908 (2021). This paper reports the use of a high-specificity AsCas12a enzyme.

    Article  ADS  CAS  PubMed  PubMed Central  Google Scholar 

  4. Kao, T. et al. GAPTrap: a simple expression system for pluripotent stem cells and their derivatives. Stem Cell Rep. 7, 518–526 (2016). This paper reports the GAPDH site as a reliable site for gene expression in iPSCs.

    Article  CAS  Google Scholar 

  5. Ho, J. Y. et al. Promoter usage regulating the surface density of CAR molecules may modulate the kinetics of CAR-T cells in vivo. Mol. Ther. Methods Clin. Dev. 21, 237–246 (2021). This paper demonstrates the differential CAR responses using different promoters.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

Download references

Additional information

Publisher’s note Springer Nature remains neutral with regard to jurisdictional claims in published maps and institutional affiliations.

This is a summary of: Allen, A. G. et al. A highly efficient transgene knock-in technology in clinically relevant cell types. Nat. Biotechnol. https://doi.org/10.1038/s41587-023-01779-8 (2023).

Rights and permissions

Reprints and permissions

About this article

Check for updates. Verify currency and authenticity via CrossMark

Cite this article

Selection by essential-gene exon knock-in for the generation of efficient cell therapies. Nat Biotechnol 42, 388–389 (2024). https://doi.org/10.1038/s41587-023-01819-3

Download citation

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.1038/s41587-023-01819-3

  • Springer Nature America, Inc.

Navigation