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An Australian clinician-led research team has developed a gene-editing platform that could give doctors more control over cell therapies, making them more practical for patients with complex immune conditions.

The new approach using prime editing could allow clinicians to use existing immune-suppressing drugs as a 'control dial' for cell therapies and help corrected cells survive in the body.

The WEHI-led research, published in Blood, aims to make cell and gene therapies more feasible for people who need ongoing immunosuppression, including those with rare immune disorders, organ or stem cell transplant recipients and patients receiving cell therapies for cancer.

Cell and gene therapies often involve taking a patient's immune cells, correcting or engineering them in the lab and returning them to the body. But once those cells are back in the patient, clinicians have limited ways to selectively boost or suppress them.

This is particularly challenging for people who need ongoing immunosuppression, including some patients with rare immune disorders, transplants, severe infections or cancers linked to immune suppression.

The drugs needed to control disease or protect a transplanted organ can also suppress the therapeutic cells that the patient needs.

Co-senior author Dr. Miles Horton said the team used prime editing, an emerging gene-editing technology that can make precise DNA changes, to address a practical problem seen in clinical care.

"We've used genetic engineering to rewire these cells so clinicians could use existing drugs, already sitting in the hospital pharmacy, as a control dial," said Horton, a WEHI researcher and pediatrics resident at the Royal Children's Hospital.

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