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Researchers discuss ethics of gene editing

(Graham Jaehnig/Daily Mining Gazette) Adam Niemi posed for a picture back in August as he stepped into the role of sports editor with the Daily Mining Gazette.

HOUGHTON — Within six years, targeted gene editing has gone from a pipe dream to routine.

Michigan Technological University faculty Paul Goetsch, Caryn Heldt, and Alexandra Morrison held a panel discussion on the opportunities and hazards that presents following a screening of the documentary “Human Nature” on the opening night of the 41 North Film Festival.

Introducing genes into a cell used to be a scattershot, essentially random process. One “miracle cure” from the early 2000s treated children with a disease giving them no resistance to infection. For some children, it worked. In others, the gene was inserted into another part of the cell, causing leukemia.

Targeting changes became easier with the use of CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats). The system, discovered by scientist Francisco Mojica, is used by bacteria, which use genetic strands from viruses to repel future invasions.

Researchers can use CRISPR sequences to bind to specific sections of DNA, using it to activate or deactivate certain genes. Revolutionary in 2013, CRISPR has since spread to virtually every institution doing genetic research.

Goetsch, an assistant professor in the biological sciences department, focuses his research on the question of how one genome of DNA can create cells for specialized functions, such as skin cells or liver cells. His lab uses CRISPR, exploring the DNA of a microscopic groundworm.

“This technique is so interesting and powerful,” he said. “I’m actually going to be introducing a new CRISPR course in biological sciences to talk about the ethics and the application.”

Heldt’s lab works on gene therapy, including how to manufacture large amounts of gene therapy drugs at a reasonable price. They focus on viral gene therapies, where viruses are modified to replicate not themselves, but a gene missing in a person. Clinical trials on gene therapy for hemophiliacs have been promising, Heldt said; they lack the Factor 8 gene, which causes blood to clot. After one dose of gene therapy, people who once had to go to the hospital after incidents as minor as running into the side of a table now have enough clotting ability to go only once or twice a year for more serious cuts.

“One of the really amazing things with this particular therapy is they’re only making 10% of the factor 8 a normal person makes,” said Heldt, director of Tech’s Health Research Institute. “So it doesn’t take a lot.”

However, personalized medicine — taking cells from a person, editing their DNA as needed, and giving cells back to them — is expensive, Heldt said.

“While this is great and amazing technology, it’s not very quickly going to be for the world,” Heldt said. “It’s only going to be for the people who can afford it.”

The film did a decent job of complicated terrain, panelists said, though with caveats.

Morrison, an assistant professor of philosophy whose research includes engineering ethics, said the movie explored ambiguity through the wide range of uses, though its direct tackling of ethics questions was ham-fisted. But she would have liked to see the movie explore tools not just as something wielded by people, but as a force acting on them and affecting how they view the world.

“I don’t think they quite did that, but they definitely created a platform for audiences and communities to have that conversation,” she said.

Goetsch said the film captured the excitement among researchers at seeing research go from impossible to surprisingly cheap.

“We’re seeing the researchers be like ‘Oh, think of the things I could do with this,’ and then ‘Oh, they want to do that, that’s horrible. But me? I’m so excited about it,'” he said.

One point the movie addressed well is how ethical concerns are being addressed so early in the technology’s development, Heldt said. She compared it to Food and Drug Administration process, where regulations typically arise only after something has gone wrong.

“I think it’s really impressive that that is being brought up so early in the conversation,” she said.

Changing the DNA does not affect the genes those patients would pass on to their descendants. The panelists drew a sharp distinction between that and “germ-line editing” — changes to reproductive cells, such as eggs and sperm.

Ethics has traditionally been approached in terms of what choice to make in one manageable situation, Morrison said. But developments, such as genetic engineering or climate change, demand a longer view.

“We have to be thinking about justice in terms of intergenerational justice, and if you think of responsibility on any scale smaller than that, we’re culpable,” she said.

Goetsch took the position of the protagonist of “Human Nature,” a boy with sickle-cell anemia, who said the decision should be up to the person.

“Germline means you’re making the decision for someone you haven’t met, who has absolutely no control,” Goetsch said.

The 41 North Film Festival continues Saturday and Sunday. For a full schedule, go to 41northfilmfest.mtu.edu.

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