The 3rd International Summit on Human Genome Editing: Progress, Controversies, and Access to Therapies

Scientists point out that it is not strange that other dishonest researchers are editing the human genome in top secrecy. Eben Kilsay, a medical anthropologist at the University of Oxford, says it wouldn’t be strange if there were more children made with CRISPR-Cas9 in the years leading up to 2018. Francis Crick Institute biologist Robin Lovell-Badge points out that there is no fundamental change in the technical aspects of genome editing, which involves manipulating the genes of unborn babies. Human genome editing is a technology for which safety has not yet been secured, and even after the announcement of Haji Ankui, various potential challenges have been pointed out, such as the possibility of unintentional mutations caused by human genome editing and the risk of mosaic embryos being born with edited and unedited cells. remains.

Currently, in many countries, including the United States, no robust governance framework exists to ensure that genome-edited embryos do not implant. In February 2023, the Chinese government issued new guidelines for ethical conduct in biological and medical research. However, one expert points out that these guidelines are for hospitals, research institutes, and universities, and do not reflect the modern situation in which advanced research has become possible in the private sector. It ignores the fact that private ventures and even individuals are increasingly launching high-tech and possibly harmful research projects.

In fact, Haji Ankui said at a press conference after his release that he was conducting research on the treatment of hereditary muscular dystrophy in Hong Kong and was approaching individual investors. The bigger problem he raised is not the creation of design babies per se, but the growing number of scientific practices that are not confined to the framework of existing scientific institutions.

While embryonic human genome editing is controversial, researchers who edit genomes in somatic cells rather than germ cells are tackling the challenge of making gene therapy by genome editing widely available. In 2018, there were very few clinical trials of somatic genome editing therapy, but there are now more than 100.

However, as the price of existing gene therapy is skyrocketing, there are concerns that genome editing therapy will also be available at a price that only a few in the world can use. In fact, in November 2022, the US Food and Drug Administration (FDA) approved hemophilia gene therapy, but the price per treatment is $3.5 million, making it the most expensive medicine in the world. In 2023, US, UK and EU regulatory authorities are considering approving CRISPR-Cas9 therapy for the genetic disease sickle cell disease, but sickle cell disease patients are common in Africa, India and the Middle East, so too expensive to use for many patients. . Therefore, the 3rd International Summit on Human Genome Editing will examine how genome editing therapies can be accessed in low- and middle-income countries. Surprisingly, expanding the manufacturing capacity of an mRNA vaccine for COVID-19 has the potential to contribute to the response of facilitating access to genome-editing therapies in low- and middle-income countries. According to Nature, the structure used to deliver mRNA contained in vaccines to cells can also be used in genome editing therapy using RNA. A genome editing therapy researcher said that if it had not been for the Corona 19 epidemic, it would not have reached the current level. Related information this placecan be found in