Friday, July 13, 2018

Mind-Boggling Medicine

We have the tools to edit genes; we can, in the proper controlled circumstances, edit any genome pretty much any way we want. But to fight disease this way you need to do the editing reliably for thousands of cells inside a living organism, and that we are still just learning. Most of the techniques involve modified viruses and they are sloppy, slow, and have a poor record in actually saving lives. So this is big news:
Researchers now say they have a found a way to use electrical fields, not viruses, to deliver both gene-editing tools and new genetic material into the cell. By speeding the process, in theory a treatment could be available to patients with almost any type of cancer. “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.” . . .

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.
This has long been shown to work in a lab, so if the new technique can really produce enough of the altered T cells to be effective, it ought to work in people. And more:
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.

The technique may also hold great promise for treating H.I.V., Dr. Wherry said.

The H.I.V. virus infects T-cells. If they can be engineered so that the virus cannot enter the T-cells, a person infected with H.I.V. should not progress to AIDS. Those T-cells already infected would die, and the engineered cells would replace them.

Previous research has shown it might be possible to treat H.I.V. in this way. “But now there is a really efficient strategy to do this,” Dr. Wherry said.
The engineering here involves putting the cells in a bath containing the new gene you want and the CRISPR tool for cutting the genome at the right point, then using electiic fields to make cell membranes permeable enough for the right amount of the tools and genes flow in. Rather than trying to reason out the right conditions to make this happen, they just assigned a graduate student to keep trying it until he got it right:
It required a herculean effort by a graduate student, Theo Roth, to finally figure out the right molecular mixture of genes, gene-editing tools and electrical fields to modify T-cells without a virus. “He tested thousands of conditions,” Dr. Marson said.
The progress in our understanding and control of molecular genetics may end up being the most important development of my lifetime. But of course the techniques used for manipulating genes for therapeutic purposes, altering bacteria to make chemicals on demand, or to bring back mammoths can also be used to create custom diseases or engineer super babies. So when I say "most important," I don't just mean most beneficial.

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