Clinical progress in genome-editing technology and in vivo delivery techniques

Jennifer Khirallah; Maximilan Eimbinder; Yamin Li; Qiaobing Xu

doi:10.1016/j.tig.2022.12.001

Clinical progress in genome-editing technology and in vivo delivery techniques

Trends Genet. 2023 Mar;39(3):208-216. doi: 10.1016/j.tig.2022.12.001. Epub 2023 Jan 18.

Authors

Jennifer Khirallah¹, Maximilan Eimbinder¹, Yamin Li², Qiaobing Xu³

Affiliations

¹ Department of Biomedical Engineering, Tufts University, Medford, MA 02155, USA.
² Department of Pharmacology, State University of New York, Upstate Medical University, Syracuse, NY 13210, USA. Electronic address: Liyam@upstate.edu.
³ Department of Biomedical Engineering, Tufts University, Medford, MA 02155, USA. Electronic address: Qiaobing.xu@tufts.edu.

Abstract

There is wide interest in applying genome-editing tools to prevent, treat, and cure a variety of diseases. Since the discovery of the clustered regularly interspaced short palindromic repeat (CRISPR)/CRISPR-associated protein (Cas) systems, these techniques have been used in combination with different delivery systems to create highly efficacious treatment options. Each delivery system has its own advantages and disadvantages and is being used for various applications. With the large number of gene-editing applications being studied but very few being brought into the clinic, we review current progress in the field, specifically where genome editing has been applied in vivo and in the clinic, and identify current challenges and areas of future growth.

Keywords: CRISPR/Cas9; clinical trials; delivery systems; genome editing; in vivo.

Publication types

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

MeSH terms

CRISPR-Cas Systems*
Gene Editing* / methods
Treatment Outcome

Grants and funding

UG3 TR002636/TR/NCATS NIH HHS/United States