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T-cells with green fluorescent label. Photo: Alex Marson Lab

Some of the most promising cancer therapies alter the DNA of T-cells so they will attack cancerous cells. Scientists announced earlier this week in Nature that they developed an alternative process that doesn't use viruses to accomplish this and could lead to safer, more precise treatments for cancer and other diseases.

"This could be a faster, cheaper, better way of making the next generation of cell therapies."
— Alexander Marson, study author, UCSF associate professor of microbiology and immunology

Background: Gene therapy aims to reprogram genes in the immune system's T-cells that can play a role in both causing and fighting diseases. Traditional methods use viral vectors (viruses stripped of their infectious parts) to take the edited DNA across the cell membrane for gene therapy or cancer immunotherapy (such as creating CAR-T cells, which were first approved last August).

  • However, study author Theo Roth says it takes considerable time and resources to make clinical-grade viruses and issues caused by the sometimes imprecise insertion of edited genes can cause serious side effects. On top of that, there is a backlog in developing those viral vectors.
"We wanted to see if we could insert new instructions into T-cells but without the need for a viral vector."
— Theo Roth, UCSF student pursuing MD and PhD degrees, who led the studies

What they found: The researchers were able to briefly shock cells with electricity (called electroporation) to make the cell membranes more permeable to DNA edited with CRISPR-Cas9, Roth says.

Another benefit is that while viral vectors may take up to a year to build, the new method may only take a couple weeks. And, the method seems to allow longer strands of edited DNA to be inserted with more precision, which opens up the possibility of using CRISPR technology to treat diseases that require longer edited strands.

Validating the findings: The research team demonstrated the method in two settings: one checked the ability to rapidly correct an inherited genetic alteration in T-cells, and the other replaced the T-cell receptor so it recognized cancer cells.

The concerns: Gene editing is touted as having great potential, but some recent studies have demonstrated it could cause more harm than good. While this new method could "potentially improve safety," the study states, more testing needs to be done.

  • “There will have to be discussions with regulatory agencies,” another study author Kevan Herold, endocrinologist and immunologist at Yale University, told the Washington Post. “All of us are aware of the potential pitfalls here” and researchers need to answer a “critical first question: Are these cells safe to be put back into people?”

What's next: Marson tells Axios they are close to going through the regulatory process to test the method in human trials. He says they believe the method could be used for infectious diseases, autoimmune disorders and a variety of cancers.

Go deeper

Arizona Republicans censure Cindy McCain and GOP governor

Combination images of Cindy McCain and Gov. Doug Ducey. Photo: FilmMagic/FilmMagic for U.S.VETS/Michael Brochstein/SOPA Images/LightRocket via Getty Images

Arizona Republican Party members voted on Saturday to censure prominent GOP figures Cindy McCain, Gov. Doug Ducey and former Sen. Jeff Flake (R-Ariz.), who've all faced clashes with former President Trump.

Why it matters: Although the resolution is symbolic, this move plus the re-election of the Trump-endorsed Kelli Ward as state GOP chair shows the strong hold the former president has on the party in Arizona, despite President Biden winning the state in the 2020 election.

Updated 5 hours ago - Politics & Policy

Coronavirus dashboard

Illustration: Eniola Odetunde/Axios

  1. Health: Most vulnerable Americans aren't getting enough vaccine information — Fauci says Trump administration's lack of facts on COVID "very likely" cost lives.
  2. Education: Schools face an uphill battle to reopen during the pandemic.
  3. Vaccine: Florida requiring proof of residency to get vaccine — CDC extends interval between vaccine doses for exceptional cases.
  4. World: Hong Kong puts tens of thousands on lockdown as cases surge — Pfizer to supply 40 million vaccine doses to lower-income countries — Brazil begins distributing AstraZeneca vaccine.
  5. Sports: 2021 Tokyo Olympics hang in the balance.
  6. 🎧 Podcast: Carbon Health's CEO on unsticking the vaccine bottleneck.

DOJ: Capitol rioter threatened to "assassinate" Alexandria Ocasio-Cortez

Supporters of former President Trump storm the U.S. Captiol on Jan. 6. Photo: Kent Nishimura / Los Angeles Times via Getty Images

A Texas man who has been charged with storming the U.S. Capitol in the deadly Jan. 6 siege posted death threats against Rep. Alexandria Ocasio-Cortez (D-N.Y.), the Department of Justice said.

The big picture: Garret Miller faces five charges in connection to the riot by supporters of former President Trump, including violent entry and disorderly conduct on Capitol grounds and making threats. According to court documents, Miller posted violent threats online the day of the siege, including tweeting “Assassinate AOC.”

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