Non-Viral Delivery To Enable Genome Editing

Yuan Rui; David R Wilson; Jordan J Green

doi:10.1016/j.tibtech.2018.08.010

Non-Viral Delivery To Enable Genome Editing

Trends Biotechnol. 2019 Mar;37(3):281-293. doi: 10.1016/j.tibtech.2018.08.010. Epub 2018 Sep 29.

Authors

Yuan Rui¹, David R Wilson², Jordan J Green³

Affiliations

¹ Department of Biomedical Engineering, Johns Hopkins University School of Medicine, Baltimore, MD 21231, USA; Translational Tissue Engineering Center, Johns Hopkins University School of Medicine, Baltimore, MD 21231, USA; These authors contributed equally.
² Department of Biomedical Engineering, Johns Hopkins University School of Medicine, Baltimore, MD 21231, USA; Translational Tissue Engineering Center, Johns Hopkins University School of Medicine, Baltimore, MD 21231, USA; Institute for Nanobiotechnology, Johns Hopkins University School of Medicine, Baltimore, MD 21231, USA; These authors contributed equally.
³ Department of Biomedical Engineering, Johns Hopkins University School of Medicine, Baltimore, MD 21231, USA; Translational Tissue Engineering Center, Johns Hopkins University School of Medicine, Baltimore, MD 21231, USA; Institute for Nanobiotechnology, Johns Hopkins University School of Medicine, Baltimore, MD 21231, USA; Departments of Materials Science and Engineering and Chemical and Biomolecular Engineering, Johns Hopkins University, Baltimore, MD 21231, USA; Departments of Ophthalmology, Oncology, and Neurosurgery, Johns Hopkins University School of Medicine, Baltimore, MD 21231, USA; Bloomberg-Kimmel Institute for Cancer Immunotherapy, Johns Hopkins University School of Medicine, Baltimore, MD 21231, USA. Electronic address: green@jhu.edu.

Abstract

Genome-editing technologies such as zinc-finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENS), and the clustered regularly interspaced short palindromic repeat (CRISPR)-associated protein system have revolutionized biological research. Each biotechnology consists of a DNA-binding protein that can be programmed to recognize and initiate double-strand breaks (DSBs) for site-specific gene modification. These technologies have the potential to be harnessed to cure diseases caused by aberrant gene expression. To be successful therapeutically, their functionality depends on their safe and efficient delivery into the cell nucleus. This review discusses the challenges in the delivery of genome-editing tools, and highlights recent innovations in non-viral delivery that have potential to overcome these limitations and advance the translation of genome editing towards patient care.

Keywords: CRISPR/Cas; TALEN; ZFN; gene editing; non-viral.

Publication types

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

MeSH terms

Drug Delivery Systems / methods*
Drug Delivery Systems / trends
Gene Editing / methods*
Genetic Therapy / methods*
Genome, Human
Humans

Abstract

Publication types

MeSH terms

Grants and funding