Therapeutic in vivo delivery of gene editing agents

Aditya Raguram; Samagya Banskota; David R Liu

doi:10.1016/j.cell.2022.03.045

Therapeutic in vivo delivery of gene editing agents

Cell. 2022 Jul 21;185(15):2806-2827. doi: 10.1016/j.cell.2022.03.045. Epub 2022 Jul 6.

Authors

Aditya Raguram¹, Samagya Banskota¹, David R Liu²

Affiliations

¹ Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of MIT and Harvard, Cambridge, MA, USA; Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA, USA; Howard Hughes Medical Institute, Harvard University, Cambridge, MA, USA.
² Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of MIT and Harvard, Cambridge, MA, USA; Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA, USA; Howard Hughes Medical Institute, Harvard University, Cambridge, MA, USA. Electronic address: drliu@fas.harvard.edu.

Abstract

In vivo gene editing therapies offer the potential to treat the root causes of many genetic diseases. Realizing the promise of therapeutic in vivo gene editing requires the ability to safely and efficiently deliver gene editing agents to relevant organs and tissues in vivo. Here, we review current delivery technologies that have been used to enable therapeutic in vivo gene editing, including viral vectors, lipid nanoparticles, and virus-like particles. Since no single delivery modality is likely to be appropriate for every possible application, we compare the benefits and drawbacks of each method and highlight opportunities for future improvements.

Publication types

Review
Research Support, U.S. Gov't, Non-P.H.S.
Research Support, N.I.H., Extramural
Research Support, Non-U.S. Gov't

MeSH terms

CRISPR-Cas Systems / genetics
Gene Editing* / methods
Genetic Therapy / methods
Genetic Vectors
Liposomes
Nanoparticles*

Substances

Lipid Nanoparticles
Liposomes

Abstract

Publication types

MeSH terms

Substances

Grants and funding