Dive Brief:
- A report published in Protein and Cells confirms rumors that Chinese researchers have successfully used a gene-editing technique on non-viable embryos.
- They modified the gene responsible for beta-thalassaemia, a blood disorder that is potentially fatal.
- The gene-editing technique they used is known as CRISPR/Cas9.
Dive Insight:
While the efforts of the Chinese researchers were successful in vitro, they have warned that they discovered serious obstacles to using their method in medical applications. Of course, the thought process is that if this technique can be perfected, it could be used to eradicate genetic diseases in utero. But many feel that there are potential ethical challenges.
During the process, the team injected 86 embryos, waited 48 hours, and then tested them to see which had been successfully spliced. Overall, of the 54 that were genetically tested (out of the 71 that survived), only 28 were successfully spliced—and only a fraction of those actually contained the replacement genetic material.
The goal should be to be as close to 100% as possible. Despite these early problems, this should not be considered a failure, but rather an important first step.