"Over the years the unthinkable hasbecome thinkable and today we sense we are close to being able to alter humanheredity œ#)." These were the words of David Baltimore of the CaliforniaInstitute of Technology, on December 1st, when he opened a three-day meeting inWashington to discuss the morality and use of human gene editing. Dr Baltimoreis an old hand at these sorts of discussions, for he was also a participant inthe Asilomar conference, in 1975, which brought scientists together to discussa safe way of using the then-new tcchnology of recombinant DNA, and whoserecommendations influenced a generation of biotechnology researchers.
Four decades on, the need for a similarsort ofchin-waghas arisen. The InternationalSummit on Human Gene Editing has been held by the national scientific academiesof three countries — America, Britain and China. They are particularlyconcerned about whether gene editing should be used to make heritable changesto the human germ line, something Dr Baltimore described as a deep andtroubling question. Like those of Asilomar, the conclusions of this meetingwill not be binding. But the hope is that, again like Asilomar, a mixture ofcommon sense and peer pressure will create a world in which scientists aretrusted to regulate themselves, rather than having politicians and civilservants do it for them. The meeting is being held against a backdrop of rapidscientific advance, Since 2012 research into a new, easy-to-use editing tool calledCRISPR-Cas9 has blossomed. This technique involves a piece of RNA (a chemicalmessenger, which can be used to recognise a target section of DNA) and anenzyme (酶)called a nuclease that can snip unwanted genes out and paste new ones in.
Public interest was aroused in April,when Chinese scientists announced they had edited genes in non-viable ( 无活力的) humanembryos, and again in November when British researchers said they hadsuccessfully treated a one-year-old girl who had leukaemia ( 白血病),using gene-edited T-cells. T-cells are part of the immune system that attack,among other things, tumour cells. The researchers altered T-cells from ahealthy donor to encourage them to recognise and kill the patient's cancer, tomake them immune to her leukaemia drug, and to ensure they did not attack herhealthy cells.
In another recent development, a firmcalled Edit as Medicine, which is based in Cambridge, Massachusetts, has saidit hopes, in 2017, to start human clinical trials of CRISPR-Cas9 as a treatmentfor a rare genetic form of blindness known as Leber congenital amaurosis (伯氏先天性黑蒙).Though other companies are already testing gene-editing therapies, these employolder, clunkier forms of the technology that seem likely to have lesscommercial potential. Moreover, researchers at the Broad Institute, also inCambridge, said this week that they had made changes to CRISPR-Cas9 whichgreatly reduce the rate of editing errors — one of the main obstacles to thetechnique's medical use.
On the subject of germ-line editing,Eric Lander, the Broad's head, told the meeting it would be useful only in rarecases and said it might be a good idea to "exercise caution? before makingpermanent changes to the gene pool. The need for caution is advice that mightalso be heeded by those pursuing work in animals other than people, and inplants — subjects not being covered by the summit.
