For the first time, scientists have found a way to efficiently and precisely remove genes from white blood cells of the immune system and to substitute beneficial replacements, all in far less time than it normally takes to edit genes.
If the technique can be replicated in other labs, experts said, it may open up profound new possibilities for treating an array of diseases, including cancer, infections like H.I.V. and autoimmune conditions like lupus and rheumatoid arthritis.
The new work, published on Wednesday in the journal Nature, “is a major advance,” said Dr. John Wherry, director of the Institute of Immunology at the University of Pennsylvania, who was not involved in the study.
But because the technique is so new, no patients have yet been treated with white blood cells engineered with it.
….“What takes months or even a year may now take a couple weeks using this new technology,” said Fred Ramsdell, vice president of research at the Parker Institute for Cancer Immunotherapy in San Francisco. “If you are a cancer patient, weeks versus months could make a huge difference.”
“I think it’s going to be a huge breakthrough,” he added.
The Parker Institute already is working with the authors of the new paper, led by Dr. Alexander Marson, scientific director of biomedicine at the Innovative Genomics Institute — a partnership between University of California, San Francisco and the University of California, Berkeley — to make engineered cells to treat a variety of cancers.
In the new study, Dr. Marson and his colleagues engineered T-cells to recognize human melanoma cells. In mice carrying the human cancer cells, the modified T-cells went right to the cancer, attacking it.
The researchers also corrected — in the lab — the T-cells of three children with a rare mutation that caused autoimmune diseases. The plan now is to return these corrected cells to the children, where they should function normally and suppress the defective immune cells, curing the children.