Biomedical research and human applications

We are interested in the ways in which genome editing might lead to benefits for human health. We are interested in research and how knowledge and methodologies are being developed through the use of genome editing that can lead to new treatments or approaches to the prevention or avoidance of disease (whether or not those treatments, themselves, involve genome editing).

We are interested in the possibility of germ line modification, an area that has excited considerable commentary since the advantages of the CRISPR-Cas9 system were first described. However, we do not want to allow the level of discussion of germ line modification to obscure ethical issues that arise in relation to other applications, such as cell-based therapies for genetic and complex diseases, or the revived prospects of xenotransplantation. Moreover, we want to consider the proper context in which to evaluate the pursuit of these ‘high tech’ strategies and ‘high ambition’ clinical objectives in relation to possible alternatives and opportunity costs.

We are interested in the translation from research into treatment and whether genome editing raises any special considerations, either about the assessment or management of risk, or about who should assess the safety and acceptability of therapeutic use. We would like to examine, for example, whether genome editing requires different decisions to be made or other decision makers to be involved, compared to the introduction of other medical treatments.

We are interested in the fitness and preparedness of regulatory systems and the variation in regulatory provisions among different countries. We would like to examine the importance attached to global consensus and the prospects of reaching and sustaining it. We are particularly interested in who is framing the global debate and who should be involved in such discussions, and we are interested in the consequences of demurral or fragmentation of governance.

Indicative questions

The following questions are indicative of the current interests of the Working Group. We encourage you to answer as few or as many questions as you wish, and feel free to ignore those that do not relate to your own knowledge or interests. We do not expect anyone responding to address all of them. Please feel free to provide other information that you think may assist the working group and, especially, to indicate other questions that you think we ought to address but may have omitted.Current research

  • What is the current state of the art in the field? What are the current technical limitations and constraints/ bottlenecks?
  • What are the main directions of travel? What are the envisaged endpoints/ applications?
  • What is the rate of travel? What are the expected timescales for realising the envisaged endpoints?

Conditions of research and innovation

  • What are the main ‘drivers’ and ‘obstacles’ in relation to envisaged endpoints?
  • What bearing do international ethical debates and agreements (e.g. high level statements or calls for moratoria) have on the pace or organisation of research?
  • Who should lead and who should be involved in setting policy for research and human applications of genome editing?  Is this significantly different from other kinds of experimental or reproductive medicine?


  • Have advances in genome editing affected what research is funded, what research strategies are used (e.g. derivation of stem cells) or the comparative development of therapeutic strategies?
  • What are the significant decisions that need to be taken before therapeutic use of genome editing may be contemplated (for non-heritable and heritable genetic changes) and who should have the responsibility for those decisions?
  • Are the benefits and costs of treatments that involve genome editing likely to be distributed equitably (or any more or less equitably than existing or alternative treatments)? In what way might genome editing differentially affect the interests of people in vulnerable or marginalised groups?
  • What other important questions should or might we have asked in this section?

Find out more about this call for evidence and how to respond

Previous work

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