Scientists have figured out how to edit genes to prevent genetic disorders in embryos, and the discovery is simultaneously receiving applause and skepticism from Delaware scientists.
Eric Kmiec, the head of the Gene Editing Institute for Christiana Care Health System’s Helen F. Graham Cancer Center, said it raises some ethical concerns and questions.
“When you create something and change something at the start of a human life, essentially you have introduced that change into the germline, which means every descendent of that child will have that change,” Kmiec said.
And once that’s done, that genetic change is permanent, Kmiec said.
“While the intention is to repair a faulty gene that causes disease, at the end of the day you don’t know what else you’ve done,” he said.
Christiana Care’s gene editing institute is focused on using gene editing as part of a treatment for lung cancer or melanoma, to help reduce the burden of chemotherapy in patients.
Gene editing has advanced in the last five years because of a tool called a Clustered Regularly Interspaced Short Palindromic Repeats. The CRISPR, Kmiec said, is basically molecular scissors that can help change genes.
At Christiana Care, they use a CRISPR in gene editing to knock out a gene that might control the ability of a tumor cell. This helps decrease resistance to chemotherapy, Kmiec said.
And the announcement that scientists have found a way to edit a gene to remove a genetic disorder trait in embryos demonstrated that the CRISPR technology can help fight genetic-based disease, said Ron Feinberg, the medical director at Reproductive Associates of Delaware.
“In and of itself, this research only demonstrated proof-of-concept for the CRISPR technology as applied to embryos, and there was no intent to utilize these embryos for creating pregnancies,” Feinberg said.
One of his ethical concerns is if these techniques could create “genetically-modified humans.”
“Although the potential for this seems far off into the future, ethical debate should continue,” Feinberg said.
At Reproductive Associates of Delaware, a team of doctors counsel patients about potential genetic risk to their offspring, but Feinberg said for them, identifying a significant genetic disease risk to a future child is fairly rare.
“Fortunately, the very large majority of known genetic disease is due to recessive mutations, meaning that both parents need to 'carry' the mutation,” Feinberg said.
As the scientific and medical community waits to hear of any more advancements in gene editing, Kmiec said the science is make big strides, sooner rather than later.
“Gene editing will change medicine. If you can do something at the gene level, you begin to get at the cause of the disease,” he said.