Natural anti-AIDS genetic editing baby is born, causing panic

On November 26, He Jiankui, a scientist from Shenzhen Southern University of Science and Technology in China, announced the day before the 2nd International Human Genome Editor Summit that a pair of genetically edited babies named Lulu and Nana were born in China in November. However, once this news was reported, the results of genetic editing were unpredictable, and whether ethical issues were resolved, etc. caused widespread controversy, and once it was on the microblogging hot search list.

According to reports, the genes of the twins have been modified to make them naturally resistant to AIDS after birth. This is the world's first genetically edited baby that is immune to AIDS. It also means that China has made a historic breakthrough in the field of disease prevention in gene editing technology.

Gene editing - an effective treatment

According to He Jiankui, genetic editing surgery is one step more than conventional IVF, that is, during the fertilized egg period, the Cas9 protein and the specific guiding sequence are injected into the needle with a needle of 5 micrometers and about one-twentieth of the hair. The fertilized egg of the cell. His team uses the "CRISPR/Cas9" gene editing technology, which precisely locates and modifies genes, also known as "gene scalpels."

This genetic surgery modified the CCR5 gene, which is one of the major co-receptors of the HIV virus invading the body cells. Previous data show that about 10% of people in the Nordic population have a natural CCR5 gene deletion. People with this mutation can close the most pathogenic HIV infection, so that the virus can not invade human cells, which can naturally infect the HIV virus.

He Jiankui will also present experimental data of his project team in mice, monkeys and human embryos at the summit. In the sequencing of 50 human embryos, no off-target was found; in all human normal embryos, more than 44% of embryos were edited. He Jiankui also showed the results of the umbilical cord blood test of the genetic surgery baby, which proved that the genetic surgery was successful and did not find off-target phenomenon. He said that the results still require time to observe and test, so a 18-year follow-up plan was prepared.

Since its inception, CRISPR/Cas9 technology has attracted attention from scientists around the world for its simple, high-efficiency research in different fields such as medicine, animal and plant breeding, and drug screening. He Jiankui stressed: "For a small number of families, genetic surgery is a new hope to cure hereditary diseases and prevent serious diseases." George Church, a professor of genetics at Harvard Medical School and a well-known expert in genetic engineering, said: "Considering HIV There is an expanding trend toward global public health threats. I think He Jiankui has chosen a very good target gene."

Can genetic editing stand on the ethical footsteps?

Not long ago, a research team at Sun Yat-sen University released the first domestic comparative report on gene editorial cognition for the general public and HIV carriers. Over 60% of respondents were positive about the use of gene editing technology. A total of 575 HIV carrier questionnaires showed that 94.78% of HIV carriers supported gene editing techniques for HIV prevention. According to a survey conducted by the Pew Research Center in the United States in April 2018 for 2,537 American adults, 60% of Americans support genetic editing of unborn babies, arguing that genetic editing is one to reduce the risk of serious illness. An effective medical tool.

In conducting scientific research, He Jiankui took the lead in proposing the “core values” to be followed in the field of genetic technology research and application, including “Mercy for families in need” for the groups that really need it, and only for serious diseases. "Respect a child's autonomy" on the premise of respect for the child's autonomy, "life needs to struggle" (Genes do not define you), "Everyone deserves freedom from genetic disease" and other five ethical principles.

According to an expert who had served as a member of the Medical Ethics Committee, according to previous practice, clinical research such as genetic editing and other technologies does not require reporting to the health department, but only the medical ethics committee in the hospital. Just fine. According to the relevant website disclosure, this clinical trial has been approved by the ethics committee of Shenzhen and the Women and Children's Hospital, and has been registered at the China Clinical Trial Registration Center. However, some insiders said that Shenzhen and the Women's and Children's Hospital are the Putian Department Hospital.

After the incident was reported, Shenzhen and the US Women and Children Hospital responded that the hospital was related to the matter and the hospital did not receive relevant information. Moreover, it is reported that the relevant person in charge of the Medical Ethics Expert Committee of the Shenzhen Municipal Health and Family Planning Commission told the reporter that it had not received the ethical review report of the project. Whether the ethical approval of this clinical trial is true remains questionable.

Is genetic editing safe?

After the incident, more than 100 Chinese scientists issued a joint statement, expressing any firm opposition and strong condemnation of any attempt to rashly attempt to make hereditary human embryonic gene editing without strict ethical and safety review at this stage.

In addition to being questioned about ethical review issues, the clinical trial was also questioned by peer experts: Is CCR5 an optimal target for gene editing and is it necessary to do so?

How does Cas9 nuclease and sgRNA affect embryo development? Is the endonuclease activity of Cas9 toxic to embryos? These questions require scientists to conduct detailed research at the molecular level or its functional level, and animal models are needed to study the effects of genetic editing on multiple generations and to explore whether the descendants of genome editing are healthy and normal.

The main reason why genetic editing has not been promoted is that the medical profession cannot predict its potential harm, and the technical level is still immature. Problems such as genetic recombination, off-target effects, genomic mutations, and increased sensitivity of the disease may not be solved.

However, because the current information about infants has not been confirmed, whether genetic health problems can be confirmed at one time, whether genetic editing treatment is safe and effective is still a key issue. Is it a violation of ethical rules or opens the magic door to save humanity?

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