The rapid development of the powerful new CRISPR-Cas9 gene-editing technology raises as many questions as it answers. Now a group of leading research organisations has thrown open the discussion to public debate. Is it now time, they ask, to the use these techniques to treat patients by altering the genes of reproductive cells and embryos to tackle genetic disease?
The CRISPR-Cas9 gene editing system is a form of "molecular scissors". It allows very precise modification of DNA sequences and so raises the prospect of accurate correction of faults in genes that can cause crippling genetic disease. While gene editing has been possible for many years, the new approach, piggybacking on natural methods used by bacterial cells, offers a much more efficient and targeted technology.
Work on applying the technique to non-reproductive cells is already in progress, with research programmes focused on HIV, sickle-cell disease and haemophilia. It is also being investigated in tackling cancer, where immune cells could be modified to attack the affected cells. More contentious, however, is its application to human reproductive cells and embryos, because the changes would then be passed down to future generations. Modification of these germline cells is strictly controlled in many countries and any change will require extensive public debate.
The UK’s position is relatively progressive compared to some other western countries. The Human Fertilisation and Embryology Act (you can see an unofficial amended text here) allows limited use of gene editing technologies on early stage human embryos for research purposes. This can only be done under licence and cannot involve embryos over 14 days old. But moving into clinical treatment of disease through modification of germline cells is not currently permitted.
The initial joint statement issued today by five leading UK research funders (Academy of Medical Science (AMS), the Association of Medical Research Charities (AMRC), the Biotechnology and Biological Sciences Research Council (BBSRC), the Medical Research Council (MRC) and the Wellcome Trust) says that 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. Active early engagement with a wide range of global stakeholders will therefore be needed, which should include, but not be limited to, biomedical and social scientists, ethicists, healthcare professionals, research funders, regulators, affected patients and their families, and the wider public."
In its press release, the Wellcome Trust calls for "widespread discussion" of the issue.
Katherine Littler, the Wellcome Trust’s Senior Policy Advisor summarises the position well, saying:
“As with any emerging technology, the potential for genome editing to be applied as a therapeutic tool in future deserves careful consideration. It’s essential that we start these discussions early, by engaging in an open and inclusive debate involving scientists, ethicists, doctors, regulators, patients and their families, and the wider public.”