What’s Happening in Genome Editing Right Now?

In light of recent advances in gene-editing research and technologies, the National Academies of Sciences, Engineering, and Medicine (NASEM) launched an initiative in December 2015 to facilitate decision making for the responsible use of human gene-editing research. This initiative examined the clinical, ethical, legal and social implications of human gene editing. Their efforts included an international summit, a comprehensive study with a diverse committee of experts, and a series of public meetings for the committee to hear from different groups including patients, community leaders and policy makers.

NHGRI uses the term "genome editing" to describe techniques used to modify DNA in the genome. Other groups also use the term "gene editing". In general, these terms are used interchangeably.

International Summit

Along with the academies, the British Royal Society and the Chinese Academy of Sciences co-hosted the International Summit on Human Gene Editing. The summit lasted three days and included panels that touched upon a range of topics related to gene editing: the history of gene editing, the scientific background of gene editing and CRISPR in particular, the clinical applications of gene editing, germline modifications, somatic cell therapy, societal implications, international perspectives, governance, and issues related to equity and access to technology.

On February 14, 2017, the committee published a report of their study findings titled, Human Genome Editing: Science, Ethics, and Governance. Based on input from public meetings and stakeholders in and out of the scientific community, the report identified seven overarching principles to guide the research and clinical use of genome editing technologies. Most notably, the report concluded that while current regulations are sufficient to oversee the use of genome editing in basic research and somatic therapy trials, there are still safety, technical, and ethical issues barring the wide application of this technology in germline therapy trials. The committee recommended a set of very strict criteria to permit clinical trials for germline therapy. The report stated gene therapy trials should be limited to treatment and prevention of disease or disability, but also suggested there should be more public discussion on the permissibility of gene therapy for enhancement purposes.

The Recombinant DNA Advisory Committee (RAC) provides the National Institutes of Health (NIH) with recommendations on basic and clinical research involving recombinant DNA (in which genes from different sources are combined). On June 21-22, 2016, the RAC met to review a series of human gene transfer protocols. Among the protocols was one that proposed the use of CRISPR to edit the T cells (a type of immune cell) of patients suffering from some types of advanced cancers, namely myeloma, melanoma and sarcoma. It would be one of the first studies in the US to use CRISPR in humans. 1,2

Because CRISPR is an important tool for basic genomics research, NHGRI currently conducts and funds research that uses CRISPR technology. For instance:

• NHGRI's Embryonic Stem Cell and Transgenic Mouse Core uses CRISPR to generate laboratory mice with specific gene alterations for the study of human genetic diseases. Many NHGRI researchers take advantage of this resource.
• The Knockout Mouse Project (KOMP) is an National Institutes of Health (NIH) Common Fund initiative that NHGRI helped develop. It was designed to generate mice containing mutations in every gene of the mouse genome. For KOMP2, researchers are using CRISPR to create these mice more efficiently. Any researcher can purchase these mice for their research.
• Dr. Shawn Burgess' lab at NHGRI has developed a high-throughput pipeline that can create multiple, simultaneous gene knockouts using CRISPR/Cas9 in zebrafish (Varshney, et al., 2015). The lab has also created a database called CRISPRz that catalogs CRISPR targets in zebrafish. These target DNA sequences can be used by other researchers to identify areas of interest for their research.

[1] The first study to use CRISPR in humans took place in October 2016 in a clinical trial by a team from Sichuan University in China:

Cyranoski, D. CRISPR gene-editing tested in a person for the first time. Nature, November 15, 2016. [Full Text]

[2] Reardon, S. First CRISPR clinical trial gets green light from US panel. Nature News, June 22, 2016. [Full Text]