The first gene editing therapy in human body was implemented in the United States?

Brian Madeux, a 4-year-old American man, carries a node that changes human history. According to the Associated Press, on June 165438+ 10 13, Ma De, who suffered from Hunter syndrome, received a bold treatment: in-vivo gene editing at Benioff Children's Hospital of the University of California, San Francisco. This is the first time in the world to treat genetic diseases through in vivo gene editing.

Ma De in casual clothes is short and fat. On the day of treatment, he wore a cap to cover his head, which was raised by illness. It seems that Ma De was just lying in the hospital bed and received an ordinary infusion for three hours, but the transparent liquid flowing into Ma De's vein from the infusion tube recorded the determination of human beings to fight incurable genetic diseases. The transparent liquid contains adeno-associated virus (AAV), zinc finger nuclease (ZFN) and normal IDS gene deleted from Desheng Ma, which will do unprecedented work.

Unlike gene therapy such as CAR-T, which takes out patients' cells for blood transfusion, or uses AAV to introduce cDNA to express functional proteins in vivo, Ma De's treatment is more thorough this time, and his genome information will be permanently changed.

After a month, there will be a preliminary answer to whether the treatment is effective. After three months, the safety and effectiveness of this therapy will be further confirmed.

It is worth mentioning that the gene editing tool used by Ma De in this treatment is zinc finger nuclease, not the "gene magic scissors" CRISPR of the fire. Compared with "epigenetics" CRISPR, scientists' research on zinc finger nuclease can be traced back to 1980s, and it is considered as the first generation of gene editing tools. Although the operation of zinc finger nuclease is more complicated, the accuracy of gene editing is higher because of the longer sequence. The US Food and Drug Administration (FDA) has approved the use of zinc finger nuclease in human clinical trials, but CRISPR has not been allowed.

Made is the first human clinical trial being studied by Sangamo Therapeutics (USA) with the code of "SB-9 13". Before the experiment, it has been approved by the relevant advisory committee of the National Institutes of Health (NIH).

Saint-Garmen Company has obtained a series of key patents of zinc finger nuclease, and it plans to expand the sample of this clinical trial and test it on more adult patients, so as to finally achieve the purpose of treating children patients. Although gene editing can help Ma De solve the root cause, the damage he has caused in the past 40 years is irreparable, so it is more effective to intervene from childhood.