(Bloomberg) — Chinese researchers safely treated a man with leukemia and HIV using gene-edited stem cells, a step forward in a field that was shaken last year when another Chinese scientist used the same technology to create the world’s first genetically-edited babies.
The man’s medical case, published Wednesday in the New England Journal of Medicine, is the first detailed report in a major academic journal of how doctors are using the experimental tool Crispr to manipulate the DNA of a living patient in an effort to cure disease. But even before the earlier controversy in China, there’s been a heavy note of caution in the field about how far and fast to proceed with the technology.
The patient’s dual diseases — HIV and cancer — gave researchers at the Peking University Stem Cell Research Center in Beijing an opening. The man needed a transplant of stem cells to replace the damaged ones that were causing his blood cancer. That procedure also gave them the opportunity to re-engineer a gene called CCR5 in the donor cells to be resistant to HIV.
“This is a green light for the whole field of gene editing,” Carl June, a pioneer in the use of gene therapy to treat cancer and HIV at the University of Pennsylvania, said in an interview. He published a companion piece in the journal.
The work has some parallels to the highly controversial effort by scientist He Jiankui to alter the DNA in two embryos to make the babies resistant to HIV. That effort sparked an international backlash and calls to put a moratorium on using Crispr to create permanent changes in a subject’s DNA, especially in an embryo. The latest effort is a far more incremental but legitimate effort, especially given the imprimatur of one of the world’s foremost academic journals.