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Genetic Engineering HIV and AIDS

World’s First Gene Edited Babies?

2 weeks, 4 days ago

2714  0
Posted on Nov 30, 2018, 6 p.m.

Scientist He Jiankui claims to have helped to create the world’s first genetically edited babies, twin girls which were said to have been born this month; whose DNA he says he altered with powerful gene editing tools; if his claims are true this would be a profound leap of both science and ethics.

Although this kind of work is banned in the USA, the US scientist said he took part in this work in China. This kind of work is currently banned in many countries as the DNA changes can pass to future generations and possibly risks harming other genes. As a result many mainstream scientists thinks it’s too unsafe to risk trying and some have denounced this report as human experimentation.

Embryos from 7 couples were said to be altered by the scientist during fertility treatments in China, which have thus far resulted in one pregnancy; claiming his goal was not to cure or prevent inherited disease, rather to try to bestow a trait and ability to resist possible future infection with HIV/AIDS.

The parents of the twin girls declined to be identified or interviewed, and there is no independent confirmation of his claims such as being published in a journal to be vetted by other experts. The scientist revealed his claims in Hong Kong to an organizer of an international conference on gene editing, and an exclusive interview with The Associated Press.

The scientist says he felt a strong responsibility that it was not just to make a first but make it an example, and society will decide what to do next in terms of allowing or forbidding such science. Many other scientists were astounded to hear his claims with some strongly condemning it and others interested to hear more, especially in terms of gene editing for HIV.

Scientists have discovered how to edit genes using CRISPR-cas9 technology to supply a needed gene or disable one that is causing problems; only recently has this been tried in adults to treat diseases and changes are confined to that person. Editing embryos, sperm, or eggs is a whole other thing, these changes can be inherited. Some feel it is too premature to try, others feel it is justifiable. The USA does not allow this kind of work except for lab research; while China outlaws human cloning but not specifically gene editing.

He Jiankui states that he practiced gene editing in mice, monkey, and human embryos in lab trials for several years and has applied for patents in his methods. Embryo gene editing for HIV was chosen because these infections are a common in China, this work sought to disable CCR5 genes that form a protein doorway that allows HIV virus to enter a cell.

Couples were recruited through a Beijing based AIDS advocacy group: all male participants in his project had HIV and all of the women did not, but the editing was not aimed at preventing the risk of transmission, as males had infection deeply suppressed and there are other ways to keep their infection from infecting offspring other than altering genes: couples affected by HIV were offered a chance to have a child that might be protected from a similar fate of infection.

Gene editing occurred during IVF, first sperm was washed, a single clean sperm was placed into a single egg to create an embryo, then the gene editing tool was added. At 3-5 days a few cells were removed from the embryos to check for editing. Couples decided whether to edit or unedit embryos for pregnancy attempts, 16 of 22 embryos were edited with 11 embryos being used in 6 implant attempts before the twin pregnancy was achieved.

After testing one of the twins is suggested to have both copies of the intended altered gene, while the other twin has just one, with no evidence of harm to other genes. Those with one copy of the gene can still get infected with HIV, however some research suggests their health might decline more slowly if they do.

Since then several scientists have reviewed the material that He Jiankui provided to the AP, tests so far are suggested to be insufficient to say editing worked to rule out harm, noting evidence of editing being incomplete, and at least one twin appears to be a patchwork of cells with various changes, almost as if not editing at all.

Dr. Kiran Musunuru of the University of Pennsylvania and Harvard University’s George Church have questioned the decision to allow one of the copies to be used in pregnancy attempt, as the researchers new in advance that both copies of the intended gene had not been altered saying in this child almost nothing will be gained in terms of protection exposing it to all of the unknown safety risks; and use of that embryo suggests main emphasis was on testing editing rather than avoiding disease.

Musunuru explains that even if gene editing worked perfectly people without normal CCR5 genes faces higher risks of getting other viruses such as West Nile and dying from the flu. It is not clear whether the participants fully understood purpose and potential risks and benefits. He Jiankui says he made the goals clear and informed them of risks and that this was never tried before; He also said he would provide insurance coverage for any child conceived through the project until they reach 18 and longer if they agree once they are adults.

Further attempts have been put on hold until the safety of this one are analyzed and experts weigh in. The scientist has sought for and received approval for this project from Shenzhen Harmonicare Women’s and Children’s Hospital, which was not one of the 4 hospitals that was provided embryos for his research or pregnancy attempts. Staff at other hospitals were kept in the dark about the nature of his work to keep some of the participants information from being disclosed other than the fact that the samples might contain HIV.

Currently He Jiankui is under investigation by government bodies and by his own university. His work has not been yet been independently confirmed, but regulators have swiftly condemned his experiments. The National Health Commission has ordered local officials to investigate his actions. If the births are confirmed the case will be handled in accordance to relevant laws and regulations, it is not clear whether there could be possible criminal charges. Thus far the response within China has been to condemn and criticize his work. The scientist also faces probes by the Shenzhen City Medical Ethics Expert Board and the Chinese Academy of Science’s academic division.

Southern University of Science and Technology of China in Shenzhen said in a statement they were not informed of the scientist’s human gene editing work and have also opened an investigation as this has seriously violated its academic ethics and standards. He Jiankui has been on leave but remains on the faculty and has a lab at the school. Questions remain about the way he proceeded, giving official notice of his work long after he said he started it on Nov 8 on a Chinese registry of clinical trials.

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https://www.apnews.com/4997bb7aa36c45449b488e19ac83e86d

https://apnews.com/2ba2279ca61740afaebebf83bf6f0243

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