After feeling ill, a 27 year old patient in China checked himself into the hospital and found out not only did he have AIDS, but he also had lymphoblastic leukemia. After receiving such diagnostics you can imagine the inner turmoil that transpired within the young man, but doctors offered him a tiny ray of hope. He would be eligible to participate in a trial that allows CRISPR to be used on an HIV patient for the first time.
The trial would purge the patients existing bone marrow from his body and replace them with millions of stem cells donated by a healthy donor. The cells were first edited to disrupt a gene called CCR5, the gene HIV uses to infiltrate your immune cells, then infused in the patient. The stem cells would hopefully place the patients cancer in remission, and in addition, suppress HIV in the patients cells.
After more than two years later, the patients cancer is in full remission. The gene edited cells have not only survived, but are also keeping his body supplied with the adequate amount of blood and immune cells. The patient however still has AIDS.
While this experiment did not cure the patients of AIDS, it shows that CRISPR is safe to use with humans. “The safety profile appears to be acceptable” writes Carl H. June, M.D. in an article. “The engineered stem cells did not appear to be immunogenic in this patient.” That is the stem cells did not provoke an immune response. He also claims that no off-target effects of the genome editing were detected, a problem that many scientist worry about word wide. So the first use of CRISPR on an HIV patient seems to be a success.
Once thought virtually impossible to cure, scientist settled on attempting to use drugs to control the disease. But then in 2007, a doctor took a patient with HIV/AIDS and infused him with blood cells from a donor with a natural mutation of the CCR5 gene called CCR5 Delta-32 and he became the first person thought cured of the disease. Since then scientist has been one the search for a way to genetically disable the CCR5 gene. CRISPR seems a viable way to achieve that goal.
Using CRISPR to edit the genes of people still remains an hot button issue globally. China however, does not seem to shy away from pushing up against those boundaries. Last year, a Chinese scientist named He Jiankui shocked the world when he announced that he had used CRISPR to edit human embryos. It remains to be seen how these provocations will effect the debate.