Medical science 2017, Karolinska Institutet

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“Of course we are aware that this is a charged issue. But we also feel that we have a great deal of support from patients.”

Photo:Ulf Sirbom.

in the special cells we have chosen”. What does she think the long-term benefit of CRISPR will be? “Alongside research, I am involved in an organisation that works with public health in East Africa and I think about what this technology can mean for lowincome countries. A cheap and easily available tool such as CRISPR provides more opportunities for these countries’ researchers to conduct good research. And in discussions about CRISPR, both improved crops and the opportunity to fight malaria have been mentioned. It would be fantastic if CRISPR is able to save people from dying of hunger and tropical diseases”. IN ANOTHER PART OF Karolinska Institutet, the Department of Clinical Sciences, Intervention and Technology, Division of Obstetrics and Gynaecology, Fredrik Lanner and his research group have just begun to use CRISPR. Their research involves the first few days of human embryo development. At this stage the embryo is called a blastocyst and is a microscopic clump of cells. The aim of the research is basic science: understanding more about what regulates the embryo’s development. Fredrik Lanner hopes that this knowledge will contribute to improved infertility treatment in future. “Fifteen per cent of all couples who want to have children have some form of fertility problem. So the more we know about normal embryonic development, the better we will be able to understand the causes of various fertility problems,” he says. Fredrik Lanner is planning to use CRISPR in the same way as Giulia Gaudenzi and many other researchers; in order to disable the function of a gene and see what difference this makes. But the fact that his research involves human embryos makes this a special case. “Embryonic stem cells and altering the genome are each individually research areas that are subject to ethical discussion – and we combine them. Of course we are aware that this is a charged issue. But we also feel that we

have a great deal of support from the patients. Close to 80 percent of patients who have undergone IVF treatment that we ask choose to donate their left over embryos to this research, embryos that would otherwise be destroyed,” he says. One benefit of conducting embryorelated research in Sweden is that the legislation is straightforward and clear, says Fredrik Lanner. “In Sweden, following strict ethical scrutiny, it is possible to conduct research on human embryos up until the fourteenth day. They must then be destroyed.We conduct research up to day seven, before the embryo would normally have attached to the wall of the uterus. At this stage it is no larger than a grain of sand and consists of about 200 cells.” In April 2015, a Chinese research group became the first in the world to report CRISPRmodified human embryos. This experiment was performed on defective IVF embryos that would not otherwise have developed into

Background: Major interest in the world 2127 of research Number of academic articles in which CRISPR is mentioned. Source: PubMed

CRISPR IS PREDICTED TO BECOME

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602 281 126 2012

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children, but it was still much discussed. “Their study led to much debate, and to people pushing for a general ban on all CRISPR research on human embryos. It is good that this is being discussed, but a ban would be very unfortunate,” says Fredrik Lanner. The argument in favour of a general ban is to prevent genetic changes being made that would be inherited by future generations. Such interventions are already banned in Sweden and many other countries, but not all. Fredrik Lanner himself thinks that development of CRISPR will not go venture that way in any case. The most likely reason for daring to make genetic changes to your child would be to prevent the child from developing a genetic disease that you yourself have”. There is already a much simpler and safer method that is used for this purpose: PGD, preimplantation genetic diagnosis. PGD involves the testing of IVF embryos prior to implantation so as to avoid implanting embryos that are predisposed to a disease. “It is thus currently technically possible to choose embryos on the basis of any characteristic we known the genetics of,” Fredrik Lanner points out. “Nevertheless, PGD is only used for avoiding serious diseases, not to choose gender or eye colour or anything else. Society has put in place rules for this activity and they work. I think that society will be able to deal with the issue of how CRISPR should be used just as well”.

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In August 2012, the researchers Emmanuelle Charpentier, Umeå University, and Jennifer Doudna, University of California, published the idea that the CRISPR-Cas9 complex could be used as a genetic technology. There has been rapid development since then and CRISPR-Cas9 has been adapted to work in ever more organisms – eventually even people.

significant within a range of areas: Regenerative medicine and for the development of gene therapies for genetic diseases, as well as for medical treatments within other areas such as infectious diseases and cancer. On top of this there are applications outside of biomedicine: The production of crops that provide greater harvests and are more resistant to drought and pests, modified microorganisms for everything from bioenergy to the purification of emissions, genetically modified mosquitoes that stop spreading diseases. The expectations linked to this new genetic tool are quite simply enormous. “The only limit is your imagination”, as one researcher put it when interviewed by Radio Sweden. Medical Science–2017

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