New genome-editing techniques are so specific, efficient and versatile that old arguments ruling out genetic modification in humans as too unreliable and unsafe to consider may no longer apply, said Group Leader Robin Lovell-Badge in introducing a new event series at the Francis Crick Institute.
Robin's talk at the inaugural 'Medicine at the Crick' event gave an introduction to genome editing methods and how they could potentially be used to treat patients with genetic conditions in the future.
It has been possible to manipulate DNA sequences in bacteria since the 1970s, he explained, and every new technology - from the first IVF baby in 1978 and Dolly the sheep in 1997 to the 2006 creation of induced pluripotent stem cells - has prompted debate about the possibility of genetically modifying humans, with concerns of designer babies or a new form of eugenics. "Previously it has been possible to say that these methods are too inefficient, too unsafe to use," Robin said.
Precise and efficient DNA editing
With the advent of genome-editing techniques, such as CRISPR-Cas9, that can alter DNA sequences precisely and efficiently, Robin wondered if the debate is changing. There is the prospect that genome editing could be used to edit the germline, preventing the inheritance of rare and severe genetic conditions by subsequent generations. "The methods are still not safe to use, more research is needed," said Robin. "However, it seems inevitable that they will be made to work efficiently and probably safely."
A panel discussion later in the event considered some of the ethical issues involved in the use of genome editing to treat genetic disorders in patients.
Other talks reported the latest in gene therapy and genome editing in clinical studies that don't involve altering the germline. Here, stem cells from patients' bone marrow is taken, gene therapies are used in the lab, and the cells transferred back to the patients.
The potential of genome editing for treating patients with sickle cell disease was outlined by Matthew Porteus of Stanford University in his keynote lecture. Claire Booth of the Institute for Child Health at UCL and Great Ormond Street Hospital described how her group has been using gene therapy to treat young children with severe immune deficiency disorders.
Bridging science and clinical practice
The Medicine at the Crick events are designed to bring lab-based scientists together with clinicians to consider the latest advances in biomedicine and how they might impact the treatment of patients.
Peter Ratcliffe, Director of Clinical Research at the Crick, explained that the events are part of the Crick's efforts to bridge "the boundary between science and clinical practice". By providing a place where discussion can take place, the aim is to stimulate new ideas, collaborations and bring developments closer to the point where they can benefit patients.
Peter highlighted three new schemes at the institute for clinicians early in their careers who wish to do science and develop laboratory research skills: there are doctoral fellowships, postdoctoral career development fellowships and group leader positions available at the Crick.
Director of the Crick Sir Paul Nurse described Medicine at the Crick as "an exciting adventure for us at the Crick". He said: "It reflects the fact that although the Crick is a discovery institute, we want to encourage interaction between those trained in medicine and clinical research and what goes on here."
The next Medicine at the Crick event is on Thursday 1 November and considers the latest of what we know about how bacteria in our gut affect our health.