Work

The Promise and Challenge of In Vivo Delivery for Genome Therapeutics

Year: 2017

Type: review

Abstract: CRISPR-based genome editing technologies are poised to enable countless new therapies to prevent, treat, or cure diseases with a genetic basis. However, the safe and effective delivery of genome editing enzymes represents a substantial challenge that must be tackled to enable the next generation of genetic therapies. In this Review, we summarize recent progress in developing enzymatic tools to combat genetic disease and examine current efforts to deliver these enzymes to the cells in need of correction. Viral vectors already in use for traditional gene therapy are being applied to enable in vivo CRISPR-based therapeutics, as are emerging technologies such as nanoparticle-based delivery of CRISPR components and direct delivery of preassembled RNA-protein complexes. Success in these areas will allow CRISPR-based genome editing therapeutics to reach their full potential.

Source: ACS Chemical Biology

DOI: 10.1021/acschembio.7b00680

Authors: Ross C. Wilson, Luke A. Gilbert

Institutions: QB3, Innovative Genomics Institute, University of California, Berkeley, University of California, San Francisco, UCSF Helen Diller Family Comprehensive Cancer Center

Language: English

Topic: CRISPR and Genetic Engineering

Subfield: Molecular Biology

Field: Biochemistry, Genetics and Molecular Biology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: green

Funders: National Institutes of Health, National Cancer Institute