We raise awareness and tackle issues with key decision makers and stakeholders
Genome editing is a powerful technology that has the potential to improve health. It allows sections of DNA from a genome to be precisely replaced or removed using “molecular scissors”.
The application of these tools is already having a game-changing effect on research intended to further our understanding of the roles of specific genes and processes in health and disease. In the future, these tools also hold the potential to be applied clinically to prevent or treat lethal and/or seriously debilitating genetic diseases. The concept of genome editing is not new: for many years, scientists have applied a range of tools to manipulate genetic sequences. However, rapid technological developments in this area – namely the emergence of the CRISPR-Cas9 system – have meant that targeted, highly efficient editing of a genome sequence may become relatively simple. This has cast a spotlight on these technologies, and, in particular, the possibility that they could be applied to enable widespread editing of human cells for therapeutic benefit.
Research using genome editing tools holds the potential to significantly progress our understanding of many key processes in biology, health and disease and for this reason we believe that responsibly conducted research of this type, which is scientifically and ethically rigorous and in line with current legal and regulatory frameworks, should be allowed to proceed.
We support the use of genome editing in preclinical biomedical research as well as studies that progress and refine these technologies. The science is still at a relatively early stage and potential therapeutic applications are not yet here but within the UK, the Human Fertilisation and Embryology Authority (HFEA) have given permission to one group of scientists, at the Francis Crick Institute, to use CRISPR-Cas9 to modify genes of embryos during the first 7 days of embryonic development. These embryos donated by women who have previously undergone IVF and have excess embryos, will need to be destroyed within 14 days, and can’t be implanted into a woman.
There may be future potential to apply genome editing in a clinical context using human germ cells or embryos, though this is prohibited by law in the UK and unlikely to be permissible in other European jurisdictions at present. This raises important ethical and regulatory questions, which need to be anticipated and explored in a timely and inclusive manner as the basic research proceeds and prior to any decisions about clinical application.
Animals in research
As a member of the Association of Medical Research Charities (AMRC) Fight for Sight supports the principle of using animals in research. We believe it is an important part of the research process to advance our understanding of eye conditions and to develop treatments, but should only be used where there is no alternative.
All AMRC member charities support this principle, as outlined in the AMRC's statement on the use of animals in research.