The gene-editing performed on Chinese twins Lula and Nana may have failed its purpose of immunizing the children from HIV and created unintended mutations, scientists said on Tuesday after the original research was made public.
Several excerpts from the manuscript were released by the MIT Review for identifying how He Jiankui, the scientist behind this experiment, ignored the scientific norms and used unethical methods on the twins. The gene-edited twins were born in late 2018, creating shockwaves in the scientific community.
Scientist He made claims that this would be a medical breakthrough and would be able to control the HIV epidemic. It was not clear whether this experiment was proven to be successful. The team had not reproduced the gene mutation that confers this resistance.
Scientific norms and unethical behavior
Several scientists have claimed that He's research and claims on the variant gene of CCR5 is inaccurate and a blatant misinterpretation of the actual data. There is only a small percent of people who are born with immunity because of a mutation in a gene called CCR5. He claimed that he had targeted this gene using the powerful tool of CRISPR. This particular tool is said to have revolutionized the field since its arrival in 2012.
While He's team targeted the right gene, scientists say that He was not able to replicate the required variation of 'Delta 35'.The scientific community says that the researchers in the project made 'off-target' edits.
Lack of access to treatments to avoid HIV in children
The scientists believe that the parents of the twins wanted to partake in this experiment for the wrong reasons. This has given rise to the ethical concerns behind this experiment. Lula and Nana's father tested positive for HIV, which carries a lot of social stigma in China. This had made it impossible for the parents to go to fertility centers and have access to treatment. There is a technique that would have prevented the infection from being passed on to the unborn children.
Jeanne O'Brien, a reproductive endocrinologist at Shady Grove Fertility told the MIT Technology Review that there are speculations that the lack of access to the fertility centers prompted the parents to be involved in the experiment despite the many risks to the children.
Where is He Jiankui?
Gene editing of the human embryo is outlawed in most parts of the world, including China and the US. One year after the gene-editing experiment was unveiled, the fate of the Chinese scientist remains unclear. Various news reports have said he has not been seen since January. China had vowed action against him even as experts and scientists had called his experiment unethical. He Jiankui has also attempted to publish his research findings in various journals but it has remained unpublished.