Date Added: 14/09/2017

Date Updated: 14/09/2017

Overview of CRISPR - genome editing technology

Specialties: Other, not otherwise specified

Technology Type: Procedures - Programmes - Other

Stage of development: Experimental - pilot or phase II

Stage of EAA: Assessment Complete

Description, patients and keywords:

Genome editing with CRISPR is a rapidly developing field that, although in its infancy, holds great promise for future clinical applications, providing a major health benefit to a diverse range of patients with diseases of a genetic origin. However, there are many issues to be considered before this technology can be introduced into clinical practice including safety and effectiveness, as well as the ethical and regulatory implications of genome editing.

It is envisaged that there will be a great demand for genome editing from affected patients and families over time. Although the first human clinical studies using CRISPR have just commenced in China and the United States, this technology should only be considered an investigational technology. Current concerns around this technology focus on the potential for, and rate of, off-target effects. It is imperative that safety and clinical effectiveness data are collected from ongoing clinical trials to inform policy and decision-making around the potential introduction of this technology into clinical practice.

This report is work in progress and should not be used for external distribution without permission from the originating agency. Users should be aware that reports are based on information available at the time of research and often on a limited literature search.