The NIH Somatic Cell Genome Editing program.

Document Type

Article

Publication Date

4-1-2021

Publication Title

Nature

Keywords

JMG

JAX Source

Nature 2020 Apr 8; 592:195-204

Volume

592

Issue

7853

First Page

195

Last Page

204

ISSN

1476-4687

PMID

33828315

DOI

https://doi.org/10.1038/s41586-021-03191-1

Abstract

The move from reading to writing the human genome offers new opportunities to improve human health. The United States National Institutes of Health (NIH) Somatic Cell Genome Editing (SCGE) Consortium aims to accelerate the development of safer and more-effective methods to edit the genomes of disease-relevant somatic cells in patients, even in tissues that are difficult to reach. Here we discuss the consortium's plans to develop and benchmark approaches to induce and measure genome modifications, and to define downstream functional consequences of genome editing within human cells. Central to this effort is a rigorous and innovative approach that requires validation of the technology through third-party testing in small and large animals. New genome editors, delivery technologies and methods for tracking edited cells in vivo, as well as newly developed animal models and human biological systems, will be assembled-along with validated datasets-into an SCGE Toolkit, which will be disseminated widely to the biomedical research community. We visualize this toolkit-and the knowledge generated by its applications-as a means to accelerate the clinical development of new therapies for a wide range of conditions.

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