Format

Send to

Choose Destination
Expert Opin Drug Deliv. 2018 Sep;15(9):905-913. doi: 10.1080/17425247.2018.1517746. Epub 2018 Sep 12.

Delivery approaches for CRISPR/Cas9 therapeutics in vivo: advances and challenges.

Author information

1
a Department of Chemistry , University of Massachusetts Amherst , Amherst , Massachusetts , USA.
2
b School of Chemical and Biotechnology , Sastra Deemed-to-be University,Tirumalaisamudram , Thanjavur , Tamil Nadu , India.

Abstract

Therapeutic gene editing is becoming a viable biomedical tool with the emergence of the CRISPR/Cas9 system. CRISPR-based technologies have promise as a therapeutic platform for many human genetic diseases previously considered untreatable, providing a flexible approach to high-fidelity gene editing. For many diseases, such as sickle-cell disease and beta thalassemia, curative therapy may already be on the horizon, with CRISPR-based clinical trials slated for the next few years. Translation of CRISPR-based therapy to in vivo application however, is no small feat, and major hurdles remain for efficacious use of the CRISPR/Cas9 system in clinical contexts. Areas covered: In this topical review, we highlight recent advances to in vivo delivery of the CRISPR/Cas9 system using various packaging formats, including viral, mRNA, plasmid, and protein-based approaches. We also discuss some of the barriers which have yet to be overcome for successful translation of this technology. Expert opinion: This review focuses on the challenges to efficacy for various delivery formats, with specific emphasis on overcoming these challenges through the development of carrier vehicles for transient approaches to CRISPR/Cas9 delivery in vivo.

KEYWORDS:

CRISPR/Cas9; gene therapy; in vivo genome editing; intracellular delivery; nonviral delivery; therapeutic strategies; viral delivery

PMID:
30169977
PMCID:
PMC6295289
[Available on 2019-09-12]
DOI:
10.1080/17425247.2018.1517746
[Indexed for MEDLINE]

Supplemental Content

Full text links

Icon for Taylor & Francis
Loading ...
Support Center