Common bacterial resistance or barriers to new gene therapy January 17, 2018 Source: Science Network Golden Staphylococcus aureus Image Source: Pasieka Getty Images According to a new study to be published in a peer-reviewed journal, a gene-editing therapy that is very popular in the laboratory may trigger an immune response when used in humans. However, it is not yet certain how much this will cause serious problems in new gene therapy, which is designed to prevent diseases caused by defective genes. "The biggest question will be: How does it affect treatment?" said Amy Wagers, a stem cell biologist at Harvard University and the Gasline Diabetes Center who did not participate in the study. She said that in rats, this gene editing tool triggers an immune response, but it is still safe and effective. However, no one knows what will happen to people. “This requires research,†she said. The CRISPR-Cas9 system, which is used as a "genetic scissors" for DNA modification, is usually derived from Staphylococcus aureus or Streptococcus pyogenes. “Many people have been exposed to these two pathogens by the time they reach the year, and their bodies may remember both bacteria and thereby respond to them after re-contacting them.†Stanford University led the study. Child scientist and stem cell biologist Matt Porteus said. Related results were recently published on the preprinted server bioRxiv. Previous exposures may invalidate genetic editing because the body quickly clears all CRISPR-Cas9 proteins. Or worse, it may turn the “derailment†phenomenon that has existed in this field for more than 10 years into an immune storm. The related problem has caused the death of a young gene therapy patient, Jesse Gelsinger, in 1999. Since the relevant research results in 2013, the virus-edited genetic tools have been highly regarded in the biomedical field. “We are sharing everyone’s excitement about developing Cas9 gene editing, but we also need to learn from the lessons learned from gene therapy, rather than ignoring the possibility of problems.†Porteus said, “all of us are considering Cas9-based Gene therapy, so this potential problem should be carefully considered." (Jin Nan)
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Common bacterial resistance or barriers to new gene therapy