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A gene-editing therapy, which is popular in the lab, can trigger an immune response when used in humans, according to a new study to be published in a peer-reviewed journal.
but it is not yet possible to determine how serious this will cause new gene therapy, which aims to stop diseases caused by defective genes.
"'s big question will be: what impact will it have on treatment? Amy Wagers, a stem cell biologist at Harvard University and the Gaslin Diabetes Center who was not involved in the study, said. In rats, the gene-editing tool triggers an immune response, but is still safe and effective,
.
, however, no one knows what will happen to people.
" this needs to be studied.
," she said.
the CRISPR-Cas9 system, a "gene scissor" that is used as a modified DNA, usually derived from Staphylococcus aureus or streptococcus.
"Many people are exposed to both bacteria by reaching adulthood, and their bodies may remember both bacteria and thus immune them to re-exposure to them."
" said Matt Porteus, a pediatric scientist and stem cell biologist who led the study at Stanford University.
results were recently published on bioRxiv, a preprinted server.
previous exposure may invalidate gene editing, as the body will soon remove all CRISPR-Cas9 proteins.
or worse, it could turn an immune storm that has been around for more than a decade, leading to the death of a young gene therapy patient, Jesse Gelsinger, in 1999.
the virus-edited genetic tool has been highly regarded in the biomedical field since the results of the 2013 test were blowing out. "We're sharing the excitement of doing Cas9 gene editing, but we're also learning the lessons of gene therapy, not ignoring the possibility of a problem,"
.
"We are all thinking about Cas9-based gene therapy, so we should seriously consider this potential problem," Porteus said.
"